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Gene Therapy Market

Gene Therapy Market by Type of Therapy, Type of Gene Delivery Method Used, Type of Vector Used, Target Therapeutic Areas, Route of Administration, and Key Geographical Regions: Industry Trends and Global Forecasts (5th Edition), 2022-2035

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Example Insights

The image provides context of Gene Therapy Market. Considering the potential of gene therapies to treat rare, inherited and acquired diseases, as well as those previously deemed incurable, there is a growing interest in evaluating the potential of such products against rare disorders This image provides list of mgene therapies marketed and currently under development. Over 1,150 gene therapy candidates are currently being investigated across different stages of development for the treatment of a myriad of disease indications; till date, 11 gene therapies have been commercialized The image provides details on the current gene therapy market landscape. More than 70% of the therapies are currently in early stages of development; close to 80% therapies being evaluated in late stage studies (phase II and above) use viral vectors for delivery of the target gene This image looks at the details of clinical trials related to gene therapies. In the last six years, more than 18,000 patients have been enrolled in registered clinical trials focused on gene therapies; ~45% of these trials are being conducted to evaluate therapies against oncological diseases
This infographic provides valuation of start-ups involved in the gene therapy domain. Start-ups are spearheading the innovation in the gene therapy domain; the valuation of a start-up is not solely dependent on profits, instead, it depends on experience and amount invested by industry stakeholders This image provides details on various initiatives taken by big pharma players related to gene therapies. In order to tap into the lucrative opportunity associated with gene therapies, big pharma players have adopted various approaches, from proprietary product development to strategic investments, to advance their portfolios This image looks at the details of patents related to gene therapies domain. Over the years, the intellectual capital related to gene therapies and gene editing has grown at a commendable pace; several patents have been filed by both industry stakeholders and academic players This image looks at the mergers and aquistions of players involved in gene therapies domain. Rising interest in this domain is reflected in the number of mergers and acquisitions reported in the last few years; 50% of such initiatives were focused on gaining access to novel platforms or therapeutic area consolidation
This infographic looks at the funding raised by players in gene therapy domain. Foreseeing lucrative returns, many public and private investors have made investments worth USD 36.4 billion, across more than 560 instances, since 2015; the funding activity is well distributed across geographical regions This image looks at key stakeholders involved in gene therapy supply chain. CMOs offering vector manufacturing services have become an integral part of the gene therapy supply chain, owing to their ability to overcome the associated challenge This infographic provides cost price analysis of gene therapies. Treatment cost is one of the major concerns in this market; we anticipate more affordable pricing and reimbursement strategies to evolve in due course This image looks at the key commercialization strategies undertaken by the stakeholders in this domain. To monetize the full potential, stakeholders are already actively exploring diverse commercialization strategies, across different stages of a product’s launch cycle
This image provides details on gene therapies market size. The market opportunity associated with gene therapies is anticipated to witness an annualized growth of nearly 18% over the next decade The image provides segmentaion of cell therapy market. In the long term, the projected opportunity is anticipated to be well distributed across different therapeutic areas, routes of administration and key geographical regions    

 

Report Description

Gene Therapy Market Overview

The gene therapy market is estimated to be worth $2.6 billion in 2023 and is expected to grow at compounded annual growth rate (CAGR) of 18.0% during the forecast period. Over the last two decades, there have been several breakthroughs related to the development of gene therapies. In 2020, Libmeldy™, an ex vivo gene therapy received approval for the treatment of metachromatic leukodystrophy. To provide more context, the treatment regimen of such therapies, encompassing gene replacement and gene-editing modalities, is aimed at correction of the mutated gene in patients using molecular carriers (viral and non-viral vectors). Further, post the onset of the COVID-19 pandemic, there has been a steady increase in the investigational new drug (IND) applications filed for cell and gene therapies. In fact, in 2021, more than 200 gene therapies were being evaluated in phase II and III studies. Moreover, in 2022, six gene therapies are expected to receive the USFDA market approval. Promising results from ongoing clinical research initiatives have encouraged government and private firms to make investments to support therapy product development initiatives in this domain. In 2021 alone, gene therapy developers raised around USD 9.5 billion in capital investments. Taking into consideration the continuous progress in this domain, gene therapies are anticipated to be used for the treatment of 1.1 million patients suffering from a myriad of disease indications, by 2035.

Presently, more than 250 companies are engaged in the development of various early and late-stage gene therapies, worldwide. In recent years, there has been a significant increase in the integration of novel technologies, such as gene modification, gene-editing, genome sequencing and manipulation technologies (molecular switches), in conjugation with gene delivery methods. For instance, the CRISPR-Cas9 based gene-editing tool is one of the remarkable technological advancements, which enables the precise alteration of the transgene. It is worth mentioning that the new generation delivery platforms, including nanoparticles and hybrid vector systems, have been demonstrated to be capable of enabling effective and safe delivery of gene based therapeutics. Further, a variety of consolidation efforts are currently ongoing in this industry. Such initiatives are primarily focused on expanding and strengthening the existing development efforts; this can be validated from the fact that 56% of the total acquisitions reported in the domain were focused on drug class consolidation. Driven by the collective and consistent efforts of developers and the growing demand for a single dose of effective therapeutic, the gene therapy market is anticipated to witness significant growth in the foreseen future.

Recent Developments in Gene Therapy Market:

Several recent developments have taken place in the field of gene therapy. We have outlined some of these recent initiatives below. These developments, even if they took place post the release of our market report, substantiate the overall market trends that have been outlined in our analysis.

  • In August 2023, Rett syndrome received fast track designation from the FDA for TSHA-102 used for the treatment of patients suffering from Rett syndrome.  
  • In August 2023, Xcell Biosciences entered into a collaboration with Labcorp to develop and validate cell and gene therapies.  
  • In May 2023, Ray Therapeutics closes USD 100 million in a Series A funding round with an aim to advance optogenetic gene therapies.

Scope of the Report

The Gene Therapy Market (5th Edition) by Type of Therapy (Gene Augmentation, Oncolytic Viral Therapy, Immunotherapy and Others), Type of Gene Delivery Method Used (Ex vivo and In vivo), Type of Vector Used (Adeno-associated Virus, Adenovirus, Herpes Simplex Virus, Lentivirus, Non-Viral Vectors, Retrovirus and Others), Target Therapeutic Areas (Cardiovascular Diseases, Dermatological Diseases, Genetic Diseases, Hematological Diseases, Infectious Diseases, Metabolic Diseases, Muscle-related Diseases, Oncological Diseases, Ophthalmic Diseases and Others), Route of Administration (Intraarticular, Intracerebral, Intracoronary, Intradermal, Intralesional, Intramuscular, Intrapleural, Intrathecal, Intratumoral, Intravenous, Intravesical, Intravitreal, Subretinal, Topical and Others), and Key Geographical Regions (US, Europe, Asia-Pacific and rest of the world): Industry Trends and Global Forecasts, 2022-2035 report features an extensive study of the current market landscape and the likely future potential associated with the gene therapy market, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, immunotherapies and gene editing therapies. Amongst other elements, the report features:

  • A detailed overview of the overall market landscape of gene therapies, including information on their phase of development (marketed, clinical, preclinical and discovery), key therapeutic areas (autoimmune diseases, cardiovascular diseases, dermatological diseases, genetic diseases, hematological diseases, hepatic diseases, immunological diseases, infectious diseases, inflammatory diseases, metabolic diseases, muscle-related diseases, neurological diseases, oncological diseases, ophthalmic diseases and others), target disease indication(s), type of vector used, type of gene / molecule targeted, type of therapy (gene augmentation, immunotherapy, oncolytic viral therapy and others), type of gene delivery method used (ex vivo and in vivo), route of administration and special drug designation(s) awarded (if any).
  • A detailed overview of the current market landscape of players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain.
  • An elaborate discussion on the various types of viral and non-viral vectors, along with information on design, manufacturing requirements, advantages and limitations of currently available gene delivery vectors.
  • A discussion on the regulatory landscape related to gene therapies across various geographies, namely North America (the US and Canada), Europe and Asia-Pacific (Australia, China, Hong Kong, Japan and South Korea), providing details related to the various challenges associated with obtaining reimbursements for gene therapies. 
  • An elaborate discussion on the various commercialization strategies that have been adopted by drug developers engaged in this domain across different stages of therapy development, including prior to drug launch, at / during drug launch and post-marketing stage.
  • Detailed profiles of marketed and late stage (phase II / III and above) gene therapies, along with information on the development timeline of the therapy, current development status, mechanism of action, affiliated technology, patent portfolio strength, dosage and manufacturing details, as well as details related to the developer company.
  • A review of the various emerging technologies and therapy development platforms that are being used to manufacture gene therapies, featuring detailed profiles of technologies that were / are being used for the development of four or more products / product candidates. 
  • An in-depth analysis of various patents that have been filed / granted related to gene therapies and gene editing therapies, since 2017, based on several relevant parameters, such as type of patent (granted patents, patent applications and others), publication year, regional applicability, CPC symbols, emerging focus areas, leading industry players (in terms of the number of patents filed / granted), and patent valuation.
  • A detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2022, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company’ product, target therapeutic area and deal multiples.
  • An analysis of the investments made at various stages, such as seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other equity offerings, by companies that are engaged in this domain.
  • An analysis of completed, ongoing and planned clinical studies, based on several relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.
  • An analysis of the various factors that are likely to influence the pricing of gene therapies, featuring different models / approaches that may be adopted by manufacturers to decide the prices of these therapies.
  • An analysis of the startup companies engaged in this domain (established between 2017-2022) based on year of experience.
  • A detailed review of the various gene therapy-based initiatives undertaken by big pharma players, highlighting trend across parameters, such as number of gene therapies under development, funding information, partnership activity and patent portfolio strength. 
  • An informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength.
  • A case study on the prevalent and emerging trends related to vector manufacturing, along with information on companies offering contract services for manufacturing vectors. The study also includes a detailed discussion on the manufacturing processes associated with various types of vectors.

A discussion on the various operating models adopted by gene therapy developers for supply chain management, highlighting the stakeholders involved, factors affecting the supply of therapeutic products and challenges encountered by developers across the different stages of the gene therapy supply chain.

One of the key objectives of the report was to estimate the existing market size and the future opportunity associated with gene therapies, over the next decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the evolution of the market for the period 2022-2035. Our year-wise projections of the current and future opportunity have further been segmented on the basis of [A] type of therapy (gene augmentation, immunotherapy, oncolytic viral therapy and others), [B] type of gene delivery method used (ex vivo and in vivo), [C] type of vector used (adeno-associated virus, adenovirus, herpes simplex virus, lentivirus, non-viral vectors, retrovirus and others), [D] target therapeutic areas (cardiovascular diseases, dermatological diseases, genetic diseases, hematological diseases, infectious diseases, metabolic diseases, muscle-related diseases, oncological diseases, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebral, intracoronary, intradermal, intralesional, intramuscular, intrapleural, intrathecal, intratumoral, intravenous, intravesical, intravitreal, subretinal, topical and others), and [F] key geographical regions (US, Europe, Asia-Pacific and rest of the world). In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industry’s growth.

The opinions and insights presented in this study were Influenced by discussions conducted with multiple stakeholders in this domain. The report features detailed transcripts of interviews held with the following individuals:

  • Buel Dan Rodgers (Founder and CEO, AAVogen)
  • Sue Washer (President and CEO, AGTC)
  • Patricia Zilliox (President and CEO, Eyevensys)
  • Christopher Reinhard (CEO and Chairman, Gene Biotherapeutics (previously known as Cardium Therapeutics))
  • Adam Rogers (CEO, Hemera Biosciences)
  • Ryo Kubota (CEO, Chairman and President, Kubota Pharmaceutical Holdings (Acucela))
  • Al Hawkins (CEO, Milo Biotechnology)
  • Jean-Phillipe Combal (CEO, Vivet Therapeutics)
  • Robert Jan Lamers (former CEO, Arthrogen)
  • Tom Wilton (former CBO, LogicBio Therapeutics)
  • Michael Triplett (former CEO, Myonexus Therapeutics)
  • Molly Cameron (former Corporate Communications Manager, Orchard Therapeutics)
  • Cedric Szpirer (Executive and Scientific Director, Delphi Genetics)
  • Marco Schmeer (Project Manager) and Tatjana Buchholz (former Marketing Manager, PlasmidFactory)
  • Jeffrey Hung (CCO, Vigene Biosciences)

All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

Frequently Asked Questions

  • Who are the key industry players engaged in the development of gene therapies? 
  • How many gene therapy candidates are present in the current development pipeline? Which key disease indications are targeted by such products? 
  • Which types of vectors are most commonly used for effective delivery of gene therapies?
  • What are the key regulatory requirements for gene therapy approval, across various geographies?
  • Which commercialization strategies are most commonly adopted by gene therapy developers, across different stages of development?
  • What are the different pricing models and reimbursement strategies currently being adopted for gene therapies?
  • What are the various technology platforms that are either available in the market or are being designed for the development of gene therapies?
  • Who are the key CMOs / CDMOs engaged in supplying viral / plasmid vectors for gene therapy development?
  • What are the key value drivers of the merger and acquisition activity in the gene therapy industry?
  • Who are the key stakeholders that have actively made investments in the gene therapy domain?
  • Which are the most active trial sites (in terms of number of clinical studies being conducted) in this domain?
  • How is the current and future market opportunity likely to be distributed across key market segments?

Frequently Asked Questions

Question 1: What is gene therapy?

Answer: Gene therapy refers to the treatment of congenital and hereditary disorders using foreign genes delivered to patients’ cells.

Question 2: How big is the gene therapy market?

Answer: The gene therapy market size is estimated to be worth $2.6 billion in 2023.

Question 3: What is the projected market growth of the gene therapy market?

Answer: The gene therapy market is expected to grow at compounded annual growth rate (CAGR) of 18% during the forecast period 2022 - 2035.

Question 4: Which are the leading drugs in the gene therapy market ?

Answer: Examples of key drugs under development in the gene therapy market (which have also been profiled in this market report; the complete list of drugs and their developers is available in the full report) include Lumevoq®, OTL-103, PTC-AADC, BMN 270, rAd-IFN/Syn3, beti-cel, eli-cel, lovo-cel, SRP-9001, EB-101, ProstAtak, D-Fi, CG0070, Vigil™-EWS Engensis, VGX-3100, Invossa™ (TG-C), VYJUVEKT, PF-06939926,. PF-06838435, PF-07055480, SPK-8011, AMT-061, VB-111, Generx ADXS-HPV, AGTC 501, LYS-SAF302, NFS-01, AG0302-COVID19, RGX-314 and Hologene.

Question 5: How many companies are engaged in the gene therapy market?

Answer: Presently, over 250 companies are engaged in the gene therapy market.

Question 6: How much money has been invested in the gene therapy market over the last few years?

Answer: Since 2015, more than USD 36 billion has been invested by industry and non-industry players in the gene therapy market.

Question 7: How many clinical trials have been conducted for evaluating novel gene therapies over the last few years?

Answer: Since 2017, over 290 clinical trials have been conducted for evaluating novel gene therapies.

Contents

Chapter Outlines

Chapter 2 provides an executive summary of the key insights captured in our research. It offers a high-level view on the current state of the market for gene therapies and its likely evolution in the short-mid term and long term.

Chapter 3 provides a general overview of gene therapies, including a discussion on their historical background. It further highlights the different types of gene therapies (namely somatic and germline therapies, and ex vivo and in vivo therapies), potential application areas of such products and route of administration of these therapeutic interventions. In addition, it provides information on the concept of gene editing, highlighting key historical milestones, applications and various techniques used for gene editing. The also chapter includes a discussion on the various advantages and disadvantages associated with gene therapies. Further, it features a brief discussion on the ethical and social concerns related to gene therapies, while highlighting future constraints and challenges related to the manufacturing and commercial viability of such product candidates.

Chapter 4 provides a general introduction to the various types of viral and non-viral gene delivery vectors. It includes a detailed discussion on the design, manufacturing requirements, advantages and limitations of currently available vectors.

Chapter 5 features a detailed discussion on the regulatory landscape related to gene therapies across various geographies, such as the US, Canada, Europe, Australia, China, Hong Kong, Japan and South Korea. Further, it highlights an emerging concept of reimbursement which was recently adopted by multiple gene therapy developers, along with a discussion on several issues associated with reimbursement of gene therapies.

Chapter 6 includes information on over 1150 gene therapies that are currently approved or are in different stages of development. It features a detailed analysis of the therapies, based on several relevant parameters, such as key therapeutic areas (autoimmune diseases, cardiovascular diseases, dermatological diseases, genetic diseases, hematological diseases, hepatic diseases, immunological diseases, infectious diseases, inflammatory diseases, metabolic diseases, muscle-related diseases, neurological diseases, oncological diseases, ophthalmic diseases and others), target disease indication(s), phase of development (marketed, clinical, preclinical and discovery), type of vector used, type of gene / molecule targeted, type of gene delivery method used (ex vivo and in vivo), type of therapy (gene augmentation, oncolytic viral therapy, immunotherapy and others), route of administration and special drug designation (if any). Further, we have presented a grid analysis of gene therapies based on phase of development, therapeutic area and type of therapy.

Chapter 7 provides a detailed overview of the current market landscape of players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain. Further, we have presented a logo landscape of product developers in North America, Europe, Asia-Pacific, and Middle East and North Africa region on the basis of company size.

Chapter 8 provides detailed profiles of marketed gene therapies. Each profile includes information about the innovator company, its product pipeline (focused on gene therapy only), development timeline of the therapy, its mechanism of action, target indication, current status of development, details related to manufacturing, dosage and sales, the company’s patent portfolio and collaborations focused on its gene therapy product / technology.

Chapter 9 features an elaborate discussion on the various strategies that have been adopted by therapy developers engaged in this domain across key commercialization stages, including prior to drug launch, during drug launch and post-launch stage. In addition, it presents an in-depth analysis of the key commercialization strategies that have been adopted by developers of gene therapies approved during the period 2015-2022.

Chapter 10 provides detailed profiles of drugs that are in advanced stages of clinical development (phase II / III and above). Each drug profile provides information on the development timeline of the therapy, current developmental status, route of administration, developers, primary target indication, special drug designation received, target gene, dosage, mechanism of action, affiliated technology, patent portfolio strength, clinical trials and collaborations (if any). 

Chapter 11 provides a list of technology platforms that are either available in the market or in the process of being designed for the development of gene therapies. In addition, it features brief profiles of some of the key technologies. Each profile features details on the various pipeline candidates that have been / are being developed using the technology, its advantages and the partnerships that have been established related to the technology platform. Further, the chapter includes detailed discussions on various novel and innovative technologies, along with brief information about key technology providers.

Chapter 12 highlights the potential target indications (segregated by therapeutic areas) that are currently the prime focus of companies developing gene therapies. These include genetic diseases, metabolic diseases, neurological diseases, oncological diseases and ophthalmic diseases. 

Chapter 13 provides an overview of the various patents that have been filed / granted related to gene therapies and gene editing therapies, since 2017, based on several relevant parameters, such as type of patent, publication year, regional applicability, CPC symbols, emerging areas and leading industry players (in terms of number of patents filed / granted). In addition, it features a competitive benchmarking analysis of the patent portfolios of leading industry players and patent valuation.

Chapter 14 features a detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2022, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company’ product, target therapeutic area and deal multiples.

Chapter 15 presents details on various funding instances, investments and grants reported within the gene therapy domain. The chapter includes information on various types of investments (such as venture capital financing, debt financing, grants, capital raised from IPO and secondary offerings) received by the companies between 2015 and 2022, highlighting the growing interest of the venture capital community and other strategic investors in this market.

Chapter 16 presents an analysis of completed, ongoing and planned clinical studies, based on several relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.

Chapter 17 highlights our views on the various factors that may be taken into consideration while deciding the price of a gene therapy. It features discussions on different pricing models / approaches, based on the size of the target population, which a pharmaceutical company may choose to adopt in order to decide the price of its proprietary products.

Chapter 18 presents a detailed analysis of the start-up companies engaged in the field of gene therapy, established between 2017-2022, based on year of experience.

Chapter 19 provides a detailed review of the various gene therapy-based initiatives undertaken by big pharma players, highlighting trend across parameters, such as number of gene therapies under development, funding information, partnership activity and patent portfolio strength. In addition, it also a detailed analysis of the big pharma players based on several parameters, such as therapeutic area, type of vector used, type of therapy and type of gene delivery method used.

Chapter 20 features an informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength.

Chapter 21 presents an elaborate market forecast analysis, highlighting the future potential of the market till the year 2035. It also includes future sales projections of gene therapies that are either marketed or in advanced stages of clinical development (phase II / III and above). Sales potential and growth opportunity were estimated based on the target patient population, likely adoption rates, existing / future competition from other drug classes and the likely price of products. The chapter also presents a detailed market segmentation on the basis of [A] type of therapy (gene augmentation, immunotherapy, oncolytic viral therapy and others), [B] type of gene delivery method used (ex vivo and in vivo), [C] type of vector used (adeno-associated virus, adenovirus, herpes simplex virus, lentivirus, non-viral vectors, retrovirus and others), [D] target therapeutic area (cardiovascular diseases, dermatological diseases, genetic diseases, hematological diseases, infectious diseases, metabolic diseases, muscle-related diseases, oncological diseases, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebral, intracoronary, intradermal, intralesional, intramuscular, intrapleural, intrathecal, intratumoral, intravenous, intravesical, intravitreal, subretinal, topical and others), and [F] key geographical regions (US, Europe, Asia-Pacific and rest of the world).

Chapter 22 provides insights on viral vector manufacturing, highlighting the steps and processes related to manufacturing and bioprocessing of vectors. In addition, it features the challenges that exist in this domain. Further, the chapter provides details on various players that offer contract manufacturing services for viral and plasmid vectors.

Chapter 23 provides a glimpse of the gene therapy supply chain. It discusses the steps for implementing a robust model and provides information related to the global regulations for supply chain. Moreover, the chapter discusses the challenges associated with supply chain of gene therapies. In addition, it features the technological solutions that can be adopted for the management of gene therapy supply chain. 

Chapter 24 summarizes the overall report, wherein we have mentioned all the key facts and figures described in the previous chapters. The chapter also highlights important evolutionary trends that were identified during the course of the study and are expected to influence the future of the gene therapy market.

Chapter 25 is a collection of interview transcripts of the discussions that were held with key stakeholders in this market. The chapter provides details of interviews held with Buel Dan Rodgers (Founder and CEO, AAVogen), Sue Washer (President and CEO, AGTC), Patricia Zilliox (President and CEO, Eyevensys), Christopher Reinhard (CEO and Chairman, Gene Biotherapeutics (previously known as Cardium Therapeutics)), Adam Rogers (CEO, Hemera Biosciences), Ryo Kubota (CEO, Chairman and President, Kubota Pharmaceutical Holdings (Acucela)), Al Hawkins (CEO, Milo Biotechnology), Jean-Phillipe Combal (CEO, Vivet Therapeutics), Robert Jan Lamers (former CEO, Arthrogen), Tom Wilton (former CBO, LogicBio Therapeutics), Michael Triplett (former CEO, Myonexus Therapeutics), Molly Cameron (former Corporate Communications Manager, Orchard Therapeutics), Cedric Szpirer (Executive and Scientific Director, Delphi Genetics), Marco Schmeer (Project Manager) and Tatjana Buchholz (former Marketing Manager, PlasmidFactory), and Jeffrey Hung (CCO, Vigene Biosciences). In addition, a brief profile of each company has been provided.

Chapter 26 is an appendix, which provides tabulated data and numbers for all the figures included in the report.

Chapter 27 is an appendix, which contains a list of companies and organizations mentioned in this report.

Table Of Contents

1. PREFACE
1.1. Scope of the Report
1.2. Research Methodology
1.3. Key Questions Answered
1.4. Chapter Outlines

2. EXECUTIVE SUMMARY
2.1. Chapter Overview

3. INTRODUCTION
3.1. Context and Background
3.2. Evolution of Gene Therapies
3.3. Classification of Gene Therapies
3.3.1. Somatic and Germline Gene Therapies
3.3.2. Ex Vivo and In Vivo Gene Therapies

3.4. Routes of Administration
3.5. Mechanism of Action
3.6. Overview of Gene Editing
3.6.1. Evolution of Genome Editing
3.6.2. Applications of Genome Editing
3.6.3. Available Genome Editing Techniques

3.7. Advantages and Disadvantages of Gene Therapies
3.7.1 Ethical and Social Concerns Related to Gene Therapies
3.7.2. Constraints and Challenges Related to Gene Therapies
3.7.3. Therapy Development Concerns
3.7.4. Manufacturing Concerns
3.7.5. Commercial Viability Concerns

4. GENE DELIVERY VECTORS
4.1. Chapter Overview
4.2. Viral and Non-Viral Methods of Gene Transfer
4.3. Viral Vectors for Genetically Modified Therapies

4.4. Types of Viral Vectors
4.4.1. Adeno-associated Viral Vectors
4.4.1.1. Overview
4.4.1.2. Design and Manufacturing
4.4.1.3. Advantages Offered
4.4.1.4. Associated Limitations

4.4.2. Adenoviral Vectors
4.4.2.1. Overview
4.4.2.2. Design and Manufacturing
4.4.2.3. Advantages Offered
4.4.2.4. Associated Limitations

4.4.3. Lentiviral Vectors
4.4.3.1. Overview
4.4.3.2. Design and Manufacturing
4.4.3.3. Advantages Offered
4.4.3.4. Associated Limitations

4.4.4. Retroviral Vectors
4.4.4.1. Overview
4.4.4.2. Design and Manufacturing
4.4.4.3. Advantages Offered
4.4.4.4. Associated Limitations

4.4.5. Other Viral Vectors
4.4.5.1. Alphavirus
4.4.5.2. Foamy Virus
4.4.5.3. Herpes Simplex Virus
4.4.5.4. Sendai Virus
4.4.5.5. Simian Virus
4.4.5.6. Vaccinia Virus

4.5. Types of Non-Viral Vectors
4.5.1. Plasmid DNA
4.5.2. Liposomes, Lipoplexes and Polyplexes
4.5.3. Oligonucleotides
4.5.4. Nanoparticles
4.5.5. Hybrid Vector Systems
4.5.6. Other Non-Viral Vectors

4.5.7. Gene Delivery Using Non-Viral Vectors
4.5.7.1. Biolistic Methods
4.5.7.2. Electroporation
4.5.7.3. Receptor Mediated Gene Delivery
4.5.7.4. Gene Activated Matrix (GAM)

5. REGULATORY LANDSCAPE AND REIMBURSEMENT SCENARIO
5.1. Chapter Overview
5.2. Regulatory Guidelines in North America
5.2.1. The US Scenario
5.2.2. Canadian Scenario

5.3. Regulatory Guidelines in Europe
5.3.1. Quality of Gene Therapy Products
5.3.2. Non-Clinical Development
5.3.3. Clinical Development

5.4. Regulatory Guidelines in Asia-Pacific
5.4.1. Chinese Scenario
5.4.1.1. Construction of DNA Expression Cassette and Gene Delivery Systems
5.4.1.2. Generation and Characterization of Cell Banks and Engineered Bacteria Banks
5.4.1.3. Manufacturing of Gene Therapy Products
5.4.1.4. Quality Control
5.4.1.5. Evaluation of Efficacy of Gene Therapy Products
5.4.1.6. Safety Evaluation of Gene Therapy Products
5.4.1.7. Clinical Trial of Gene Therapy Products
5.4.1.8. Ethics Study

5.4.2. Japanese Scenario
5.4.3. South Korean Scenario
5.4.4. Australian Scenario
5.4.5. Hong Kong Scenario

5.5. Reimbursement Scenario
5.5.1. Challenges Related to Reimbursement

5.6. Commonly Offered Payment Models for Gene Therapies

6. MARKET OVERVIEW
6.1. Chapter Overview
6.1.1. Analysis of Gene Therapy Candidates by Stage of Development

6.2. Gene Therapy Market: Clinical and Commercial Pipeline
6.2.1. Analysis by Phase of Development
6.2.2. Analysis by Therapeutic Area
6.2.3. Analysis by Type of Vector Used
6.2.4. Analysis by Type of Gene / Molecule Targeted
6.2.5. Analysis by Type of Therapy
6.2.6. Analysis by Type of Gene Delivery Method Used
6.2.7. Analysis by Route of Administration

6.3. Gene Therapy Market: Early-Stage Pipeline
6.3.1. Analysis by Phase of Development
6.3.2. Analysis by Therapeutic Area
6.3.3. Analysis by Type of Vector Used
6.3.4. Analysis by Type of Therapy
6.3.5. Analysis by Type of Gene Delivery Method Used
6.3.6. Analysis by Route of Administration

6.4. Gene Therapy Market: Special Drug Designations
6.4.1. Analysis by Special Designation(s) Awarded

6.5. Analysis by Phase of Development, Therapeutic Area and Type of Therapy (Grid Representation)

7. COMPETITIVE LANDSCAPE
7.1. Chapter Overview
7.2. Gene Therapy Market: List of Developers
7.2.1. Analysis by Year of Establishment
7.2.2. Analysis by Company Size
7.2.3. Analysis by Location of Headquarters
7.2.3.1. Analysis by Year of Establishment, Company Size and Location of Headquarters
7.2.3.2. Regional Landscape

7.3. Key Players: Analysis by Number of Pipeline Candidates

8. MARKETED GENE THERAPIES
8.1. Chapter Overview
8.2. Gendicine® (Shenzhen Sibiono GeneTech)
8.2.1. Company Overview
8.2.2. Development Timeline
8.2.3. Mechanism of Action and Vector Used
8.2.4. Target Indication(s)
8.2.5. Current Status of Development
8.2.6. Manufacturing, Dosage and Sales

8.3. Oncorine® (Shanghai Sunway Biotech)
8.3.1. Company Overview
8.3.2. Development Timeline
8.3.3. Mechanism of Action and Vector Used
8.3.4. Target Indication(s)
8.3.5. Current Status of Development
8.3.6. Manufacturing, Dosage and Sales

8.4. Rexin-G® (Epeius Biotechnologies)
8.4.1. Company Overview
8.4.2. Development Timeline
8.4.3. Mechanism of Action and Vector Used
8.4.4. Target Indication(s)
8.4.5. Current Status of Development
8.4.6. Manufacturing, Dosage and Sales

8.5. Neovasculgen® (Human Stem Cells Institute)
8.5.1. Company Overview
8.5.2. Development Timeline
8.5.3. Mechanism of Action and Vector Used
8.5.4. Target Indication(s)
8.5.5. Current Status of Development
8.5.6. Manufacturing, Dosage and Sales

8.6. Imlygic® (Amgen)
8.6.1. Company Overview
8.6.2. Development Timeline
8.6.3. Mechanism of Action and Vector Used
8.6.4. Target Indication(s)
8.6.5. Current Status of Development
8.6.6. Manufacturing, Dosage and Sales

8.7. Strimvelis® (Orchard Therapeutics)
8.7.1. Company Overview
8.7.2. Development Timeline
8.7.3. Mechanism of Action and Vector Used
8.7.4. Target Indication(s)
8.7.5. Current Status of Development
8.7.6. Manufacturing, Dosage and Sales

8.8. Luxturna™ (Spark Therapeutics)
8.8.1. Company Overview
8.8.2. Development Timeline
8.8.3. Mechanism of Action and Vector Used
8.8.4. Target Indication(s)
8.8.5. Current Status of Development
8.8.6. Manufacturing, Dosage and Sales

8.9. Zolgensma™ (Novartis)
8.9.1. Company Overview
8.9.2. Development Timeline
8.9.3. Mechanism of Action and Vector Used
8.9.4. Target Indication(s)
8.9.5. Current Status of Development
8.9.6. Manufacturing, Dosage and Sales

8.10. Collategene® (AnGes)
8.10.1. Company Overview
8.10.2. Development Timeline
8.10.3. Mechanism of Action and Vector Used
8.10.4. Target Indication(s)
8.10.5. Current Status of Development
8.10.6. Manufacturing, Dosage and Sales

8.11. Zyntelgo™ (bluebird bio)
8.11.1. Company Overview
8.11.2. Development Timeline
8.11.3. Mechanism of Action and Vector Used
8.11.4. Target Indication(s)
8.11.5. Current Status of Development
8.11.6. Manufacturing, Dosage and Sales

8.12. Libmeldy™ (Orchard Therapeutics)
8.12.1. Company Overview
8.12.2. Development Timeline
8.12.3. Mechanism of Action and Vector Used
8.12.4. Target Indication(s)
8.12.5. Current Status of Development
8.12.6. Manufacturing, Dosage and Sales

9. KEY COMMERCIALIZATION STRATEGIES
9.1. Chapter Overview
9.2. Successful Drug Launch Strategy: ROOTS Framework
9.3. Successful Drug Launch Strategy: Product Differentiation
9.4. Commonly Adopted Commercialization Strategies based on Phase of Development of Product
9.5. List of Currently Approved Gene Therapies

9.6. Key Commercialization Strategies Adopted by Gene Therapy Developers
9.6.1. Strategies Adopted Before Therapy Approval
9.6.1.1. Participation in Global Events
9.6.1.2. Collaboration with Stakeholders and Pharmaceutical Firms
9.6.1.3. Indication Expansion

9.6.2. Strategies Adopted During / Post Therapy Approval
9.6.2.1. Geographical Expansion
9.6.2.2. Participation in Global Events
9.6.2.3. Patience Assistance Programs
9.6.2.4. Awareness through Product Websites
9.6.2.5. Collaboration with Stakeholders and Pharmaceutical Firms

9.7. Concluding Remarks

10. LATE STAGE (PHASE II / III AND ABOVE) GENE THERAPIES
10.1. Chapter Overview
10.2. Lumevoq® (GS010): Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.3. OTL-103: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.4. PTC-AADC: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.5. BMN 270: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.6. rAd-IFN/Syn3: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.7. beti-cel: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.8. eli-cel: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.9. lovo-cel: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.10. SRP-9001: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.11. EB-101: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.12. ProstAtak®: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.13. D-Fi: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.14. CG0070: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.15. Vigil™-EWS: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.16. Engensis®: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.17. VGX-3100: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.18. Invossa™ (TG-C): Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.19. VYJUVEKT™: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.20. PF-06939926: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.21. PF-06838435: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.22. PF-07055480: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.23. SPK-8011: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.24. AMT-061: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.25. VB-111: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.26. Generx®: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.27. ADXS-HPV: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.28. AGTC 501: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.29. LYS-SAF302: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.30. NFS-01: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.31. AG0302-COVID19: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.32. RGX-314: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.33. Hologene 5: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results

11. EMERGING TECHNOLOGIES
11.1. Chapter Overview
11.2. Gene Editing Technologies
11.2.1. Overview
11.2.2. Applications

11.3. Emerging Gene Editing Platforms
11.3.1. CRISPR / Cas9 System
11.3.1.1. Key Components and Functions
11.3.1.2. Mechanism of Action
11.3.1.3. Targeting Efficiency and Challenges
11.3.1.4. Next-GEN CRISPR Technology
11.3.1.5. Technology Providers

11.3.2. TALENs
11.3.2.1. Structural Features
11.3.2.2. Mechanism of Action
11.3.2.3. Advantages and Challenges

11.3.3. megaTAL
11.3.3.1. Technology Providers

11.3.4. Zinc Finger Nuclease
11.3.4.1. Technology Providers

11.4. Gene Expression Regulation Technologies
11.4.1. Technology Providers

11.5. Technology Platforms for Developing / Delivering Gene Therapies

12. KEY THERAPEUTICS AREAS
12.1. Chapter Overview
12.2. Analysis by Therapeutic Area and Special Designation(s) Awarded

12.3. Oncological Diseases
12.3.1. Analysis by Target Indication
12.3.2. Analysis by Type of Vector Used

12.4. Neurological Diseases
12.4.1. Analysis by Target Indication
12.4.2. Analysis by Type of Vector Used

12.5. Ophthalmic Diseases
12.5.1. Analysis by Target Indication
12.5.2. Analysis by Type of Vector Used

12.6. Metabolic Diseases
12.6.1. Analysis by Target Indication
12.6.2. Analysis by Type of Vector Used

12.7. Genetic Diseases
12.7.1. Analysis by Target Indication
12.7.2. Analysis by Type of Vector Used

13. PATENT ANALYSIS
13.1. Chapter Overview
13.2. Gene Therapy Market: Patent Analysis
13.2.1. Scope and Methodology
13.2.1.1. Analysis by Publication Year
13.2.1.2. Analysis by Publication Year and Type of Patent
13.2.1.3. Analysis by Geography
13.2.1.4. Analysis by CPC Symbols
13.2.1.5. Analysis by Emerging Focus Areas
13.2.1.6. Leading Players: Analysis by Number of Patents
13.2.1.7. Patent Benchmark Analysis
13.2.1.8. Patent Valuation Analysis

13.3. Gene Editing Market: Patent Analysis
13.3.1. Scope and Methodology
13.3.1.1. Analysis by Publication Year
13.3.1.2. Analysis by Publication Year and Type of Patent
13.3.1.3. Analysis by Geography
13.3.1.4. Analysis by CPC Symbols
13.3.1.5. Analysis by Emerging Focus Areas
13.3.1.6. Leading Players: Analysis by Number of Patents
13.3.1.7. Patent Benchmark Analysis
13.3.1.8. Patent Valuation Analysis

13.4. Overall Intellectual Property Portfolio: Analysis by Type of Organization

14. MERGERS AND ACQUISITIONS
14.1. Chapter Overview
14.2. Merger and Acquisition Models
14.3. Gene Therapy Market: Mergers and Acquisitions
14.3.1. Analysis by Year of Merger / Acquisition
14.3.2. Analysis by Type of Agreement
14.3.3. Analysis by Geography
14.3.3.1. Continent-wise Distribution
14.3.3.2. Intercontinental and Intracontinental Deals
14.3.3.3. Local and International Deals

14.3.4. Analysis by Key Value Drivers
14.3.4.1. Analysis by Key Value Drivers and Year of Acquisition

14.3.5. Analysis by Phase of Development of the Acquired Company’s Product
14.3.6. Analysis by Therapeutic Area
14.3.7. Analysis by Deal Multiples

15. FUNDING AND INVESTMENT ANALYSIS
15.1. Chapter Overview
15.2. Types of Funding
15.3. Gene Therapy Market: Funding and Investment Analysis
15.3.1. Analysis of Funding Instances by Year
15.3.2. Analysis of Amount Invested by Year
15.3.3. Analysis of Funding Instances and Amount Invested by Type of Funding
15.3.4. Analysis of Funding Instances and Amount Invested by Year and Type of Funding
15.3.5. Analysis of Funding Instances and Amount Invested by Type of Therapy
15.3.6. Analysis of Funding Instances and Amount Invested by Geography
15.3.7. Analysis of Funding Instances and Amount Invested by Highest Phase of Development
15.3.8. Analysis of Funding Instances by Therapeutic Area
15.3.9. Most Active Players: Analysis by Number of Funding Instances and Amount Invested
15.3.10. Key Investors: Analysis by Number of Funding Instances

15.4. Concluding Remarks

16. CLINICAL TRIAL ANALYSIS
16.1. Chapter Overview
16.2. Scope and Methodology
16.3. Gene Therapy Market: Clinical Trial Analysis
16.3.1. Analysis by Trial Registration Year
16.3.2. Analysis by Trial Status
16.3.3. Analysis by Trial Phase
16.3.4. Analysis by Therapeutic Area
16.3.5. Analysis by Geography
16.3.6. Analysis by Trial Registration Year and Geography
16.3.7. Analysis by Trial Status and Geography
16.3.8. Analysis by Trial Status, Trial Phase and Geography
16.3.9. Analysis by Therapeutic Area and Geography
16.4. Analysis by Type of Sponsor
16.5. Analysis by Prominent Treatment Sites

16.6. Gene Therapy Market: Analysis of Enrolled Patient Population
16.6.1. Analysis by Trial Registration Year
16.6.2. Analysis by Trial Status
16.6.3. Analysis by Trial Phase
16.6.4. Analysis by Therapeutic Area
16.6.5. Analysis by Location of Trial Site
16.6.6. Analysis by Trial Status and Location of Trial Site
16.6.7. Analysis by Trial Status, Trial Phase and Location of Trial Site
16.6.8. Analysis by Therapeutic Area and Location of Trial Site

16.7. Concluding Remarks
16.7.1. Emerging Molecules to Watch
16.7.2. Most Important Trials to Watch

17. COST PRICE ANALYSIS
17.1. Chapter Overview
17.2. Gene Therapy Market: Factors Contributing to the Price of Gene Therapies
17.3. Gene Therapy Market: Pricing Models
17.3.1. Based on Associated Product / Component Costs
17.3.2. Based on Competition
17.3.3. Based on Patient Segment
17.3.4. Based on Opinions of Industry Experts

18. START-UP VALUATION
18.1. Chapter Overview
18.2. Valuation by Year of Experience
18.2.1. Methodology
18.2.2. Results and Interpretation

19. BIG PHARMA PLAYERS: ANALYSIS OF GENE THERAPY RELATED INITIATIVES
19.1. Chapter Overview
19.2. Gene Therapy Market: List of Most Prominent Big Pharmaceutical Players
19.2.1. Analysis by Therapeutic Area
19.2.2. Analysis by Type of Vector Used
19.2.3. Analysis by Type of Therapy
19.2.4. Analysis by Type of Gene Delivery Method

19.3. Benchmark Analysis of Key Parameters
19.3.1. Spider Web Analysis: Pipeline Strength
19.3.2. Spider Web Analysis: Merger / Acquisitions
19.3.3. Spider Web Analysis: Funding and Investments
19.3.4. Spider Web Analysis: Clinical Trials
19.3.5. Spider Web Analysis: Technologies
19.3.6. Spider Web Analysis: Patents

19.4. Benchmark Analysis of Big Pharmaceutical Players

20. DEMAND ANALYSIS
20.1. Chapter Overview
20.2. Methodology
20.3. Global Demand for Gene Therapies, 2022-2035
20.3.1. Analysis by Type of Therapy
20.3.2. Analysis by Therapeutic Area
20.3.3. Analysis by Geography

21. MARKET FORECAST AND OPPORTUNITY ANALYSIS
21.1. Chapter Overview
21.2. Scope and Limitations
21.3. Key Assumptions and Forecast Methodology
21.4. Global Gene Therapy Market, 2022-2035
21.4.1. Gene Therapy Market: Analysis by Therapeutic Area
21.4.2. Gene Therapy Market: Analysis by Type of Vector Used
21.4.3. Gene Therapy Market: Analysis by Type of Therapy
21.4.4. Gene Therapy Market: Analysis by Gene Delivery Method Used
21.4.5. Gene Therapy Market: Analysis by Route of Administration
21.4.6. Gene Therapy Market: Analysis by Geography

21.5. Gene Therapy Market: Value Creation Analysis
21.6. Gene Therapy Market: Product-wise Sales Forecasts
21.6.1. Gendicine®
21.6.1.1. Target Patient Population
21.6.1.2. Sales Forecast
21.6.1.3. Net Present Value
21.6.1.4. Value Creation Analysis

21.6.2. Oncorine®
21.6.2.1. Target Patient Population
21.6.2.2. Sales Forecast
21.6.2.3. Net Present Value
21.6.2.4. Value Creation Analysis

21.6.3. Rexin-G®
21.6.3.1. Target Patient Population
21.6.3.2. Sales Forecast
21.6.3.3. Net Present Value
21.6.3.4. Value Creation Analysis

21.6.4. Neovasculgen®
21.6.4.1. Target Patient Population
21.6.4.2. Sales Forecast
21.6.4.3. Net Present Value
21.6.4.4. Value Creation Analysis

21.6.5. Strimvelis®
21.6.5.1. Target Patient Population
21.6.5.2. Sales Forecast
21.6.5.3. Net Present Value
21.6.5.4. Value Creation Analysis

21.6.6. Imlygic®
21.6.6.1. Target Patient Population
21.6.6.2. Sales Forecast
21.6.6.3. Net Present Value
21.6.6.4. Value Creation Analysis

21.6.7. Luxturna™
21.6.7.1. Target Patient Population
21.6.7.2. Sales Forecast
21.6.7.3. Net Present Value
21.6.7.4. Value Creation Analysis

21.6.8. Zolgensma™
21.6.8.1. Target Patient Population
21.6.8.2. Sales Forecast
21.6.8.3. Net Present Value
21.6.8.4. Value Creation Analysis

21.6.9. Collategene®
21.6.9.1. Target Patient Population
21.6.9.2. Sales Forecast
21.6.9.3. Net Present Value
21.6.9.4. Value Creation Analysis

21.6.10. Libmeldy™
21.6.10.1. Target Patient Population
21.6.10.2. Sales Forecast
21.6.10.3. Net Present Value
21.6.10.4. Value Creation Analysis

21.6.11. Lumevoq® (GS010)
21.6.11.1. Target Patient Population
21.6.11.2. Sales Forecast
21.6.11.3. Net Present Value
21.6.11.4. Value Creation Analysis

21.6.12. OTL-103
21.6.12.1. Target Patient Population
21.6.12.2. Sales Forecast
21.6.12.3. Net Present Value
21.6.12.4. Value Creation Analysis

21.6.13. PTC-AADC
21.6.13.1. Target Patient Population
21.6.13.2. Sales Forecast
21.6.13.3. Net Present Value
21.6.13.4. Value Creation Analysis

21.6.14. BMN 270
21.6.14.1. Target Patient Population
21.6.14.2. Sales Forecast
21.6.14.3. Net Present Value
21.6.14.4. Value Creation Analysis

21.6.15. rAd-IFN/Syn3
21.6.15.1. Target Patient Population
21.6.15.2. Sales Forecast
21.6.15.3. Net Present Value
21.6.15.4. Value Creation Analysis

21.6.16. beti-cel
21.6.16.1. Target Patient Population
21.6.16.2. Sales Forecast
21.6.16.3. Net Present Value
21.6.16.4. Value Creation Analysis

21.6.17. eli-cel
21.6.17.1. Target Patient Population
21.6.17.2. Sales Forecast
21.6.17.3. Net Present Value
21.6.17.4. Value Creation Analysis

21.6.18. lovo-cel
21.6.18.1. Target Patient Population
21.6.18.2. Sales Forecast
21.6.18.3. Net Present Value
21.6.18.4. Value Creation Analysis

21.6.19. SRP-9001
21.6.19.1. Target Patient Population
21.6.19.2. Sales Forecast
21.6.19.3. Net Present Value
21.6.19.4. Value Creation Analysis

21.6.20. EB-101
21.6.20.1. Target Patient Population
21.6.20.2. Sales Forecast
21.6.20.3. Net Present Value
21.6.20.4. Value Creation Analysis

21.6.21. ProstAtak®
21.6.21.1. Target Patient Population
21.6.21.2. Sales Forecast
21.6.21.3. Net Present Value
21.6.21.4. Value Creation Analysis

21.6.22. D-Fi
21.6.22.1. Target Patient Population
21.6.22.2. Sales Forecast
21.6.22.3. Net Present Value
21.6.22.4. Value Creation Analysis

21.6.23. CG0070
21.6.23.1. Target Patient Population
21.6.23.2. Sales Forecast
21.6.23.3. Net Present Value
21.6.23.4. Value Creation Analysis

21.6.24. Vigil™-EWS
21.6.24.1. Target Patient Population
21.6.24.2. Sales Forecast
21.6.24.3. Net Present Value
21.6.24.4. Value Creation Analysis

21.6.25. Engensis®
21.6.25.1. Target Patient Population
21.6.25.2. Sales Forecast
21.6.25.3. Net Present Value
21.6.25.4. Value Creation Analysis

21.6.26. VGX-3100
21.6.26.1. Target Patient Population
21.6.26.2. Sales Forecast
21.6. 26.3. Net Present Value
21.6.26.4. Value Creation Analysis

21.6.27. Invossa™ (TG-C)
21.6.27.1. Target Patient Population
21.6.27.2. Sales Forecast
21.6.27.3. Net Present Value
21.6.27.4. Value Creation Analysis

21.6.28. VYJUVEKT™
21.6.28.1. Target Patient Population
21.6.28.2. Sales Forecast
21.6.28.3. Net Present Value
21.6.28.4. Value Creation Analysis

21.6.29. PF-06939926
21.6.29.1. Target Patient Population
21.6.29.2. Sales Forecast
21.6.29.3. Net Present Value
21.6.29.4. Value Creation Analysis

21.6.30. PF-06838435
21.6.30.1. Target Patient Population
21.6.30.2. Sales Forecast
21.6.30.3. Net Present Value
21.6.30.4. Value Creation Analysis

21.6.31. PF-07055480
21.6.31.1. Target Patient Population
21.6.31.2. Sales Forecast
21.6.31.3. Net Present Value
21.6.31.4. Value Creation Analysis

21.6.32. SPK-8011
21.6.32.1. Target Patient Population
21.6.32.2. Sales Forecast
21.6.32.3. Net Present Value
21.6.32.4. Value Creation Analysis

21.6.33. AMT-061
21.6.33.1. Target Patient Population
21.6.33.2. Sales Forecast
21.6.33.3. Net Present Value
21.6.33.4. Value Creation Analysis

21.6.34. VB-111
21.6.34.1. Target Patient Population
21.6.34.2. Sales Forecast
21.6.34.3. Net Present Value
21.6.34.4. Value Creation Analysis

21.6.35. Generx®
21.6.35.1. Target Patient Population
21.6.35.2. Sales Forecast
21.6.35.3. Net Present Value
21.6.35.4. Value Creation Analysis

21.6.36. AMG001
21.6.36.1. Target Patient Population
21.6.36.2. Sales Forecast
21.6.36.3. Net Present Value
21.6.36.4. Value Creation Analysis

21.6.37. OAV-101
21.6.37.1. Target Patient Population
21.6.37.2. Sales Forecast
21.6.37.3. Net Present Value
21.6.37.4. Value Creation Analysis

21.6.38. ADXS-HPV
21.6.38.1. Target Patient Population
21.6.38.2. Sales Forecast
21.6.38.3. Net Present Value
21.6.38.4. Value Creation Analysis

21.6.39. AGTC 501
21.6.39.1. Target Patient Population
21.6.39.2. Sales Forecast
21.6.39.3. Net Present Value
21.6.39.4. Value Creation Analysis

21.6.40. LYS-SAF302
21.6.40.1. Target Patient Population
21.6.40.2. Sales Forecast
21.6.40.3. Net Present Value
21.6.40.4. Value Creation Analysis

21.6.41. NFS-01
21.6.41.1. Target Patient Population
21.6.41.2. Sales Forecast
21.6.41.3. Net Present Value
21.6.41.4. Value Creation Analysis

21.6.42. AG0302-COVID19
21.6.42.1. Target Patient Population
21.6.42.2. Sales Forecast
21.6.42.3. Net Present Value
21.6.42.4. Value Creation Analysis

21.6.43. RGX-314
21.6.43.1. Target Patient Population
21.6.43.2. Sales Forecast
21.6.43.3. Net Present Value
21.6.43.4. Value Creation Analysis

21.6.44. Hologene 5
21.6.44.1. Target Patient Population
21.6.44.2. Sales Forecast
21.6.44.3. Net Present Value
21.6.44.4. Value Creation Analysis

22. VECTOR MANUFACTURING
22.1. Chapter Overview
22.2. Overview of Viral Vector Manufacturing
22.3. Viral Vector Manufacturing Process
22.3.1. Mode of Vector Production
22.3.2. Adherent and Suspension Cultures
22.3.3. Unit Processes and Multiple Parallel Processes

22.3.4. Cell Culture Systems for Production of Viral Vectors
22.3.4.1. Small Scale / Laboratory Scale Cell Culture Systems
22.3.4.2. Large Scale Cell Culture Systems
22.3.5. Culture Media Specifications

22.4. Bioprocessing of Viral Vectors
22.4.1. AAV Vector Production
22.4.2. Adenoviral Vector Production
22.4.3. Lentiviral Vector Production
22.4.4. γ -Retroviral Vector Production

22.5. Challenges Associated with Vector Manufacturing
22.6. Contract Service Providers for Viral and Plasmid Vectors

23. CASE STUDY: GENE THERAPY SUPPLY CHAIN
23.1. Chapter Overview
23.2. Overview of Gene Therapy Supply Chain
23.3. Implementation of Supply Chain Models
23.4. Logistics in Gene Therapies
23.4.1. Logistic Processes for Autologous and Allogeneic Therapies

23.5. Regulatory Supply Chain Across the Globe
23.6. Challenges Associated with Gene Therapy Supply Chain

23.7. Optimizing Cell and Advanced Therapies Supply Chain Management
23.7.1. Enterprise Manufacturing System
23.7.2. Laboratory Information Management System
23.7.3. Inventory Management System
23.7.4. Quality Management System
23.7.5. Logistics Management System
23.7.6. Patient Management System
23.7.7. Electronic Clinical Outcome Assessments System
23.7.8. Supply Chain Orchestration Platform

23.8. Recent Developments and Upcoming Trends

24. CONCLUSION
24.1. Chapter Overview

25. INTERVIEW TRANSCRIPTS
25.1. Chapter Overview
25.2. Buel Dan Rodgers (Founder and CEO, AAVogen)
25.3. Sue Washer (President and CEO, AGTC)
25.4. Patricia Zilliox (President and CEO, Eyevensys)
25.5. Christopher Reinhard (CEO and Chairman, Gene Biotherapeutics (previously known as Cardium Therapeutics))
25.6. Adam Rogers (CEO, Hemera Biosciences)
25.7. Ryo Kubota (CEO, Chairman and President, Kubota Pharmaceutical Holdings (Acucela))
25.8. Al Hawkins (CEO, Milo Biotechnology)
25.9. Jean-Phillipe Combal (CEO, Vivet Therapeutics)
25.10. Robert Jan Lamers (former CEO, Arthrogen)
25.11. Tom Wilton (former CBO, LogicBio Therapeutics)
25.12. Michael Tripletti (former CEO, Myonexus Therapeutics)
25.13. Molly Cameron (former Corporate Communications Manager, Orchard Therapeutics)
25.14. Cedric Szpirer (former Executive and Scientific Director, Delphi Genetics)
25.15. Marco Schmeer (Project Manager) and Tatjana Buchholz, (former Marketing Manager, PlasmidFactory)
25.16. Jeffrey Hung (CCO, Vigene Biosciences)

26. APPENDIX 1: TABULATED DATA

27. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS

List Of Figures

Figure 2.1 Executive Summary: Overall Market Landscape of Gene Therapies
Figure 2.2 Executive Summary: Competitive Landscape
Figure 2.3 Executive Summary: Key Therapeutic Areas
Figure 2.4 Executive Summary: Patent Analysis
Figure 2.5 Executive Summary: Mergers and Acquisitions
Figure 2.6 Executive Summary: Funding and Investment Analysis
Figure 2.7 Executive Summary: Clinical Trial Analysis
Figure 2.8 Executive Summary: Demand Analysis
Figure 2.9 Executive Summary: Market Forecast and Opportunity Analysis
Figure 3.1 Evolution of Gene Therapies
Figure 3.2 Ex Vivo Gene Therapies
Figure 3.3 In Vivo Gene Therapies
Figure 3.4 Mechanism of Action of Gene Therapies
Figure 3.5 Genome Editing: Historical Milestones
Figure 3.6 Genome Editing Techniques
Figure 3.7 Advantages and Disadvantages of Gene Therapies
Figure 4.1 Gene Transfer: Viral and Non-Viral Methods
Figure 5.1 Centralized Regulatory Pathway for Market Approval in the US
Figure 5.2 Centralized Regulatory Pathway for Market Approval in Europe
Figure 5.3 Manufacturing Requirements for Gene Therapy Products in China
Figure 5.4 Key Elements of Quality Control in China
Figure 5.5 Centralized Regulatory Pathway for Market Approval in Japan
Figure 6.1 Gene Therapies: Distribution by Stage of Development
Figure 6.2 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Phase of Development
Figure 6.3 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Therapeutic Area
Figure 6.4 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Phase of Development and Therapeutic Area
Figure 6.5 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Vector Used
Figure 6.6 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Gene / Molecule Targeted
Figure 6.7 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Therapy
Figure 6.8 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Vector Used and Type of Therapy
Figure 6.9 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Gene Delivery Method Used
Figure 6.10 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Route of Administration
Figure 6.11 Early-Stage Pipeline of Gene Therapies: Distribution by Phase of Development
Figure 6.12 Early-Stage Pipeline of Gene Therapies: Distribution by Therapeutic Area
Figure 6.13 Early-Stage Pipeline of Gene Therapies: Distribution by Phase of Development and Therapeutic Area
Figure 6.14 Early-Stage Pipeline of Gene Therapies: Distribution by Type of Vector Used
Figure 6.15 Early-Stage Pipeline of Gene Therapies: Distribution by Type of Therapy
Figure 6.16 Early-Stage Pipeline of Gene Therapies: Distribution by Type of Vector Used and Type of Therapy
Figure 6.17 Early-Stage Pipeline of Gene Therapies: Distribution by Type of Gene Delivery Method Used
Figure 6.18 Early-Stage Pipeline of Gene Therapies: Distribution by Route of Administration
Figure 6.19 Gene Therapies: Distribution by Special Designation(s) Awarded
Figure 6.20 Gene Therapies: Distribution by Phase of Development, Therapeutic Area and Type of Therapy (Grid Representation)
Figure 7.1 Gene Therapy Developers: Distribution by Year of Establishment
Figure 7.2 Gene Therapy Developers: Distribution by Company Size
Figure 7.3 Gene Therapy Developers: Distribution by Location of Headquarters
Figure 7.4 Gene Therapy Developers: Distribution by Year of Establishment, Company Size and Location of Headquarters
Figure 7.5 Gene Therapy Developers: Distribution of Players based in North America
Figure 7.6 Gene Therapy Developers: Distribution of Players based in Europe
Figure 7.7 Gene Therapy Developers: Distribution of Players based in Asia-Pacific
Figure 7.8 Gene Therapy Developers: Distribution of Players based in Middle East and North Africa
Figure 7.9 Key Players: Distribution by Number of Gene Therapies
Figure 7.10 Key Players: Distribution by Number of Gene Therapies and Phase of Development
Figure 8.1 Gendicine®: Development Timeline
Figure 8.2 Oncorine®: Development Timeline
Figure 8.3 Oncorine®: Mechanism of Action
Figure 8.4 Rexin-G®: Development Timeline
Figure 8.5 Neovasculgen®: Development Timeline
Figure 8.6 Imlygic®: Development Timeline
Figure 8.7 Imlygic®: Mechanism of Action
Figure 8.8 Strimvelis®: Development Timeline
Figure 8.9 Luxturna™: Development Timeline
Figure 8.10 Zolgensma™: Development Timeline
Figure 8.11 Collategene®: Development Timeline
Figure 8.12 Zyntelgo™: Development Timeline
Figure 8.13 Libmeldy™: Development Timeline
Figure 9.1 Successful Drug Launch Strategy: ROOTS Framework
Figure 9.2 Successful Drug Launch Strategy: Product Differentiation
Figure 9.3 Commonly Adopted Commercialization Strategies based on Phase of Development of Product
Figure 9.4 Harvey Ball Analysis: Key Commercialization Strategies Adopted by Gene Therapy Developers
Figure 9.5 Approved Gene Therapies: Historical Timeline of Geographical Expansion
Figure 9.6 Approved Gene Therapies: Snapshot of Promotional Activities on Product Websites
Figure 9.7 Key Commercialization Strategies: Harvey Ball Analysis by Ease of Implementation, Value Addition and Current Adoption
Figure 11.1 Gene Editing: Key Application Areas
Figure 11.2 Gene Editing: Emerging Technology Platforms
Figure 12.1 Gene Therapies: Distribution by Therapeutic Area and Special Designation(s) Awarded
Figure 12.2 Gene Therapies for Oncological Diseases: Distribution by Target Indication and Phase of Development
Figure 12.3 Gene Therapies for Oncological Diseases: Distribution by Type of Vector Used
Figure 12.4 Gene Therapies for Neurological Diseases: Distribution by Target Indication and Phase of Development
Figure 12.5 Gene Therapies for Neurological Diseases: Distribution by Type of Vector Used
Figure 12.6 Gene Therapies for Ophthalmic Diseases: Distribution by Target Indication and Phase of Development
Figure 12.7 Gene Therapies for Ophthalmic Diseases: Distribution by Type of Vector Used
Figure 12.8 Gene Therapies for Metabolic Diseases: Distribution by Target Indication and Phase of Development
Figure 12.9 Gene Therapies for Metabolic Diseases: Distribution by Type of Vector Used
Figure 12.10 Gene Therapies for Genetic Diseases: Distribution by Target Indication and Phase of Development
Figure 12.11 Gene Therapies for Genetic Diseases: Distribution by Type of Vector Used
Figure 13.1 Gene Therapy Patent Portfolio: Distribution by Type of Patent
Figure 13.2 Gene Therapy Patent Portfolio: Cumulative Distribution by Publication Year, 2017-2022
Figure 13.3 Gene Therapy Patent Portfolio: Distribution by Type of Patent and Publication Year
Figure 13.4 Gene Therapy Patent Portfolio: Distribution by Geography
Figure 13.5 Gene Therapy Patent Portfolio: North America Scenario
Figure 13.6 Gene Therapy Patent Portfolio: Europe Scenario
Figure 13.7 Gene Therapy Patent Portfolio: Asia-Pacific Scenario
Figure 13.8 Gene Therapy Patent Portfolio: Distribution by CPC Symbols
Figure 13.9 Gene Therapy Patent Portfolio: Emerging Focus Areas
Figure 13.10 Leading Industry Players (Gene Therapies): Distribution by Number of Patents
Figure 13.11 Gene Therapy Patent Portfolio (Sanofi and Genethon): Benchmarking by Patent Characteristics
Figure 13.12 Gene Therapy Patent Portfolio (Other Leading Industry Players): Benchmarking by Patent Characteristics
Figure 13.13 Gene Therapy Patent Portfolio: Distribution by Patent Age (2017-2022)
Figure 13.14 Gene Therapy Patent Portfolio: Valuation Analysis
Figure 13.15 Gene Editing Patent Portfolio: Distribution by Type of Patent
Figure 13.16 Gene Editing Patent Portfolio: Cumulative Distribution by Publication Year, 2017-2022
Figure 13.17 Gene Editing Patent Portfolio: Distribution by Type of Patent and Publication Year
Figure 13.18 Gene Editing Patent Portfolio: Distribution by Geography
Figure 13.19 Gene Editing Patent Portfolio: North America Scenario
Figure 13.20 Gene Editing Patent Portfolio: Europe Scenario
Figure 13.21 Gene Editing Patent Portfolio: Asia-Pacific Scenario
Figure 13.22 Gene Editing Patent Portfolio: Distribution by CPC Symbols
Figure 13.23 Gene Editing Patent Portfolio: Emerging Focus Areas
Figure 13.24 Leading Industry Players (Gene Editing): Distribution by Number of Patents
Figure 13.25 Gene Editing Patent Portfolio (Intellia Therapeutics and CRISPR Therapeutics): Benchmarking by Patent Characteristics
Figure 13.26 Gene Editing Patent Portfolio (Other Leading Industry Players): Benchmarking by Patent Characteristics
Figure 13.27 Gene Editing Patent Portfolio: Distribution by Patent Age (2017-2022)
Figure 13.28 Gene Editing Patent Portfolio: Valuation Analysis
Figure 13.29 Gene Therapy Patent Portfolio: Cumulative Distribution by Type of Organization
Figure 13.30 Gene Editing Patent Portfolio: Cumulative Distribution by Type of Organization
Figure 14.1 Mergers and Acquisitions: Cumulative Year-Wise Trend, 2015-2022
Figure 14.2 Mergers and Acquisitions: Distribution by Type of Agreement
Figure 14.3 Mergers and Acquisitions: Distribution by Year and Type of Agreement
Figure 14.4 Mergers and Acquisitions: Continent-wise Distribution
Figure 14.5 Mergers and Acquisitions: Intercontinental and Intracontinental Deals
Figure 14.6 Mergers and Acquisitions: Local and International Deals
Figure 14.7 Acquisitions: Distribution by Key Value Drivers
Figure 14.8 Acquisitions: Distribution by Year of Acquisition and Key Value Drivers
Figure 14.9 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company’s Product
Figure 14.10 Mergers and Acquisitions: Distribution by Year of Acquisition and Phase of Development of the Acquired Company’s Product
Figure 14.11 Mergers and Acquisitions: Distribution of Deal Amount by Phase of Development of the Acquired Company’s Product
Figure 14.12 Mergers and Acquisitions: Distribution by Therapeutic Area
Figure 15.1 Funding and Investments: Cumulative Distribution of Instances by Year, 2015-2022
Figure 15.2 Funding and Investments: Cumulative Distribution of Amount Invested by Year, 2015-2022 (USD Million)
Figure 15.3 Funding and Investments: Distribution of Instances by Type of Funding, 2015-2022
Figure 15.4 Funding and Investments: Distribution of Total Amount Invested by Type of Funding, 2015-2022 (USD Million)
Figure 15.5 Funding and Investments: Distribution of Instances by Year and Type of Funding, 2015-2022
Figure 15.6 Funding and Investments: Distribution of Amount Invested by Year and Type of Funding, 2015-2022 (USD Million)
Figure 15.7 Funding and Investments: Summary of Investments, 2015-2022 (USD Million)
Figure 15.8 Funding and Investments: Distribution of Instances and Amount Invested by Type of Therapy
Figure 15.9 Funding and Investments: Distribution of Instances and Amount Invested by Geography
Figure 15.10 Funding and Investments: Regional Distribution of Funding Instances
Figure 15.11 Funding and Investments: Distribution of Instances and Amount Invested by Highest Phase of Development of the Company’s Product
Figure 15.12 Funding and Investments: Distribution of Instances by Therapeutic Area
Figure 15.13 Most Active Players: Distribution by Number of Funding Instances, 2015-2022
Figure 15.14 Most Active Players: Distribution by Amount Invested, 2015-2022 (USD Million)
Figure 15.15 Funding and Investment Analysis: Distribution of Instances by Type of Investors
Figure 15.16 Leading Investors: Distribution by Number of Instances
Figure 15.17 Funding and Investment Summary, 2015-2022 (USD Million)
Figure 16.1 Clinical Trial Analysis: Distribution by Trial Registration Year, 2017-2022
Figure 16.2 Clinical Trial Analysis: Distribution by Trial Status
Figure 16.3 Clinical Trial Analysis: Distribution by Trial Phase
Figure 16.4 Clinical Trial Analysis: Distribution by Therapeutic Area
Figure 16.5 Clinical Trial Analysis: Distribution by Geography
Figure 16.6 Clinical Trial Analysis: Distribution by Trial Registration Year and Geography
Figure 16.7 Clinical Trial Analysis: Distribution by Trial Status and Geography
Figure 16.8 Clinical Trial Analysis: Distribution by Trial Status, Trial Phase and Geography
Figure 16.9 Clinical Trial Analysis: Distribution by Therapeutic Area and Geography
Figure 16.10 Clinical Trial Analysis: Distribution by Type of Sponsor
Figure 16.11 Clinical Trial Analysis: Distribution by Prominent Treatment Sites
Figure 16.12 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Registration Year, 2017-2022
Figure 16.13 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status
Figure 16.14 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Phase
Figure 16.15 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Therapeutic Area
Figure 16.16 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Location of Trial Site
Figure 16.17 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status and Location of Trial Site
Figure 16.18 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status, Trial Phase and Location of Trial Site
Figure 16.19 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Therapeutic Area and Location of Trial Site
Figure 17.1 Gene Therapy: Pricing Model Based on Patient Segment
Figure 18.1 Start-up Valuation based on Experience of the Gene Therapy Developer
Figure 19.1 Big Pharma Players: Top Pharmaceutical Companies (Heat Map Representation)
Figure 19.2 Big Pharma Players: Analysis by Therapeutic Area
Figure 19.3 Big Pharma Players: Analysis by Type of Vector Used
Figure 19.4 Big Pharma Players: Analysis by Type of Therapy
Figure 19.5 Big Pharma Players: Analysis by Type of Gene Delivery Method Used
Figure 20.1 Global Demand for Gene Therapies, 2022-2035
Figure 20.2 Demand Analysis: Distribution by Type of Therapy, 2022, 2030 and 2035
Figure 20.3 Demand Analysis: Distribution by Therapeutic Area, 2022, 2030 and 2035
Figure 20.4 Demand Analysis: Distribution by Geography, 2022, 2030 and 2035
Figure 20.5 Demand Analysis: Country-wise Distribution (Asia-Pacific), 2022, 2030 and 2035
Figure 21.1 Global Gene Therapy Market, 2022-2035 (USD Billion)
Figure 21.2 Gene Therapy Market: Distribution by Therapeutic Area, 2022, 2030 and 2035
Figure 21.3 Gene Therapy Market: Distribution by Type of Vector Used, 2022, 2030 and 2035
Figure 21.4 Gene Therapy Market: Distribution by Type of Therapy, 2022, 2030 and 2035
Figure 21.5 Gene Therapy Market: Distribution by Type of Gene Delivery Method Used, 2022, 2030 and 2035
Figure 21.6 Gene Therapy Market: Distribution by Route of Administration, 2022, 2030 and 2035
Figure 21.7 Gene Therapy Market: Distribution by Geography, 2022, 2030 and 2035
Figure 21.8 Gene Therapy Market: Country-wise Distribution (Asia-Pacific), 2022, 2030 and 2035
Figure 21. 9 Gendicine® Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.10 Oncorine® Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.11 Rexin-G® Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.12 Neovasculgen® Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.13 Strimvelis® Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.14 Imlygic® Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.15 Luxturna™ Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.16 Zolgensma™ Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.17 Collategene® Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.18 Libmeldy™ Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.19 Lumevaq® (GS010) Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.20 OTL-103 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.21 PTC-AADC Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.22 BMN 270 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.23 rAd-IFN/Syn3 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.24 beti-cel Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.25 eli-cel Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.26 lovo-cel Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.27 SRP-9001 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.28 EB-101 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.29 ProstAtak® Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.30 D-Fi Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.31 CG0070 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.32 Vigil™-EWS Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.33 Engensis® Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.34 VGX-3100 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.35 Invossa™ (TG-C) Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.36 VYJUVEKT™ Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.37 PF-06939926 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.38 PF-06838435 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.39 PF-07055480 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.40 SPK-8011 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.41 AMT-061 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.42 VB-111 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.43 Generx® Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.44 AMG001 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.45 OAV-101 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.46 ADXS-HPV Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.47 AGTC 501 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.48 LYS-SAF302 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.49 NFS-01 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.50 AG0302-COVID19 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.51 RGX-314 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.52 Hologene 5 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 22.1 Viral Vectors: Key Manufacturing Steps
Figure 23.1 Steps Involved in the Development of Gene Therapies
Figure 23.2 Overall Development Process of Cell-based Gene Therapies
Figure 23.3 Key Responsibilities of Stakeholders Involved in Advanced Therapies Supply Chain
Figure 23.4 Key Steps Involved in the Implementation of a Supply Chain Strategy
Figure 23.5 Keys Steps Involved in the Logistics Process of Autologous Therapies
Figure 23.6 Comparison of Logistics Processes for Autologous and Allogeneic Therapies
Figure 23.7 Global Regulatory Authorities for Gene Therapy Supply Chain
Figure 23.8 Overview of Supply Chain Orchestration Platform
Figure 23.9 Functions of Key Technological Solutions for Cell and Advanced Therapies Supply Chain Management
Figure 24.1 Concluding Remarks: Overall Market Overview
Figure 24.2 Concluding Remarks: Patent Analysis
Figure 24.3 Concluding Remarks: Mergers and Acquisitions
Figure 24.4 Concluding Remarks: Funding and Investments
Figure 24.5 Concluding Remarks: Clinical Trials Analysis
Figure 24.6 Concluding Remarks: Big Pharmaceutical Gene Therapy Related Initiatives
Figure 24.7 Concluding Remarks: Market Forecast and Opportunity Analysis
Figure 24.8 Concluding Remarks: Market Forecast and Opportunity Analysis

List Of Tables

Table 3.1 Comparison of Ex Vivo and In Vivo Techniques
Table 3.2 Vectors Used for Targeted Gene Delivery to Tissues / Organs
Table 3.3 Routes of Administration of Gene Therapies: Advantages and Disadvantages
Table 4.1 Viral Vectors: Key Features
Table 5.1 Approved Advanced Therapies Medicinal Products (ATMPs) and their Reimbursement Status in Europe
Table 5.2 Payment Options Available for Gene Therapies
Table 6.1 Gene Therapy Market: Clinical and Commercial Pipeline
Table 6.2 Gene Therapy Market: Early-Stage Pipeline
Table 6.3 Gene Therapy Market: Special Designation(s) Awarded
Table 7.1 Gene Therapy Market: List of Developers
Table 8.1 Gene Therapies: Marketed and Approved Products
Table 8.2 Shenzhen Sibiono GeneTech: Company Overview
Table 8.3 Gendicine®: Patent Portfolio
Table 8.4 Gendicine®: Status of Development
Table 8.5 Shanghai Sunway Biotech: Company Overview
Table 8.6 Oncorine®: Status of Development
Table 8.7 Epeius Biotechnologies: Company Overview
Table 8.8 Rexin-G®: Status of Development
Table 8.9 Human Stem Cell Institute: Company Overview
Table 8.10 Neovasculgen®: Status of Development
Table 8.11 Amgen: Company Overview
Table 8.12 Imlygic®: Status of Development
Table 8.13 Imlygic®: Recommended Dose and Schedule
Table 8.14 Imlygic®: Determination of Injection Volume based upon Lesion Size
Table 8.15 Orchard Therapeutics: Company Overview
Table 8.16 Strimvelis®: Status of Development
Table 8.17 Spark Therapeutics: Company Overview
Table 8.18 Luxturna™: Status of Development
Table 8.19 Novartis Gene Therapies: Company Overview
Table 8.20 Zolgensma™: Status of Development
Table 8.21 AnGes: Company Overview
Table 8.22 Collategene®: Status of Development
Table 8.23 bluebird bio: Company Overview
Table 8.24 Zyntelgo™: Status of Development
Table 8.25 Libmeldy™: Status of Development
Table 9.1 Libmeldy™: Presentations in Conferences Before Product Approval
Table 9.2 Luxturna™: Presentations in Conferences Before Product Approval
Table 9.3 Zolgensma™: Presentations in Conferences Before Product Approval
Table 9.4 Zyntelgo™: Presentations in Conferences Before Product Approval
Table 9.5 Imlygic®: Presentations in Conferences After Product Approval
Table 9.6 Libmeldy™: Presentations in Conferences After Product Approval
Table 9.7 Luxturna™: Presentations in Conferences After Product Approval
Table 9.8 Strimvelis™: Presentations in Conferences After Product Approval
Table 9.9 Zolgensma™: Presentations in Conferences After Product Approval
Table 9.10 Zyntelgo™: Presentations in Conferences After Product Approval
Table 9.11 Approved Gene Therapies: Patient Support Services Available on Product Websites (Comparative Analysis)
Table 10.1 Gene Therapies: Late Stage (Phase II / III and Above) Drug Candidates
Table 10.2 Lumevaq® (GS010): Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.3 OTL-103: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.4 PTC-AADC: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.5 BMN 270: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.6 rAd-IFN/Syn3: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.7 beti-cel: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.8 eli-cel: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.9 lovo-cel: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.10 SRP-9001: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.11 EB-101: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.12 ProstAtak®: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.13 D-Fi: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.14 CG0070: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.15 Vigil™-EWS: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.16 Engensis®: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.17 VGX-3100: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.18 Invossa™ (TG-C): Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.19 VYJUVEKT™: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.20 PF-06939926: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.21 PF-06838435: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.22 PF-07055480: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.23 SPK-8011: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.24 AMT-061: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.25 VB-111: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.26 Generx®: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.27 ADXS-HPV: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.28 AGTC 501: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.29 LYS-SAF302: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.30 NFS-01: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.31 AG0302-COVID19: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.32 RGX-314: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.32 Hologene 5: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 11.1 Gene Editing Technology Platforms
Table 11.2 ASC Therapeutics: Collaborations
Table 11.3 CRISPR Therapeutics: Funding Instances
Table 11.4 CRISPR Therapeutics: Collaborations
Table 11.5 Editas Medicine: Funding Instances
Table 11.6 Editas Medicine: Collaborations
Table 11.7 Emendo Biotherapeutics: Collaborations
Table 11.8 Excision Biotherapeutics: Funding Instances
Table 11.9 Intellia Therapeutics: Funding Instances
Table 11.10 Intellia Therapeutics: Collaborations
Table 11.11 Sarepta Therapeutics: Funding Instances
Table 11.12 Sarepta Therapeutics: Collaborations
Table 11.13 ToolGen: Collaborations
Table 11.14 bluebird bio: Funding Instances
Table 11.15 bluebird bio: Collaborations
Table 11.16 Sangamo Therapeutics: Funding Instances
Table 11.17 Sangamo Therapeutics: Collaborations
Table 11.18 Gene Switch Technology Platforms
Table 11.19 Precigen: Funding Instances
Table 11.20 Precigen: Collaborations
Table 11.21 MeiraGTx: Funding Instances
Table 11.22 MeiraGTx: Collaborations
Table 11.23 Gene Therapy: Technology Platforms
Table 12.1 Oncological Diseases: Information on Highest Phase of Development of Gene Therapy and Type of Vector Used
Table 12.2 Neurological Diseases: Information on Highest Phase of Development of Gene Therapy and Type of Vector Used
Table 12.3 Ophthalmic Diseases: Information on Highest Phase of Development of Gene Therapy and Type of Vector Used
Table 12.4 Metabolic Diseases: Information on Highest Phase of Development of Gene Therapy and Type of Vector Used
Table 12.5 Genetic Diseases: Information on Highest Phase of Development of Gene Therapy and Type of Vector Used
Table 13.1 Gene Therapy Patent Portfolio: List of Top CPC Symbols
Table 13.2 Gene Therapy Patent Portfolio: Summary of Benchmarking Analysis
Table 13.3 Gene Therapy Patent Portfolio: Categorizations based on Weighted Valuation Scores
Table 13.4 Gene Therapy Patent Portfolio: List of Leading Patents (in terms of Highest Relative Valuation)
Table 13.5 Gene Editing Patent Portfolio: List of Top CPC Symbols
Table 13.6 Gene Editing Patent Portfolio: Summary of Benchmarking Analysis
Table 13.7 Gene Editing Patent Portfolio: Categorizations based on Weighted Valuation Scores
Table 13.8 Gene Editing Patent Portfolio: List of Leading Patents (in terms of Highest Relative Valuation)
Table 14.1 Gene Therapy: List of Mergers and Acquisitions, 2015-2022
Table 14.2 Acquisitions: Key Value Drivers
Table 14.3 Mergers and Acquisitions: Information on Phase of Development and Therapeutic Area of the Acquired Company’s Gene Therapy Product
Table 14.4 Mergers and Acquisitions: Information on Deal Multiple based on Acquisition Amount
Table 15.1 Gene Therapy Market: Funding and Investments, 2015-2022
Table 15.2 Funding and Investments: Summary of Investments
Table 15.3 Funding and Investments: Summary of Venture Capital Funding
Table 16.1 Clinical Trial Analysis: Emerging Molecules to Watch
Table 16.2 Clinical Trial Analysis: Most Important Trials to Watch
Table 17.1 Pricing Model: Price of Marketed Gene / Cell Therapies
Table 17.2 Pricing Model: Price of Marketed Targeted Drugs (Antibody Drug Conjugates and Monoclonal Antibodies)
Table 17.3 Pricing Model: Opinions of Experts / Other Analysts
Table 18.1 Start-up Valuation: Estimates based on Overall Experience
Table 21.1 Gene Therapies: Expected Launch Years of Advanced Stage Drug Candidates
Table 21.2 Gendicine®: Target Patient Population
Table 21.3 Gendicine®: Net Present Value (USD Million)
Table 21.4 Gendicine®: Value Creation Analysis (USD Million)
Table 21.5 Oncorine®: Target Patient Population
Table 21.6 Oncorine®: Net Present Value (USD Million)
Table 21.7 Oncorine®: Value Creation Analysis (USD Million)
Table 21.8 Rexin-G®: Target Patient Population
Table 21.9 Rexin-G®: Net Present Value (USD Million)
Table 21.10 Rexin-G®: Value Creation Analysis (USD Million)
Table 21.11 Neovasculgen®: Target Patient Population
Table 21.12 Neovasculgen®: Net Present Value (USD Million)
Table 21.13 Neovasculgen®: Value Creation Analysis (USD Million)
Table 21.14 Strimvelis®: Target Patient Population
Table 21.15 Strimvelis®: Net Present Value (USD Million)
Table 21.16 Strimvelis®: Value Creation Analysis (USD Million)
Table 21.17 Imlygic®: Target Patient Population
Table 21.18 Imlygic®: Net Present Value (USD Million)
Table 21.19 Imlygic®: Value Creation Analysis (USD Million)
Table 21.20 Luxturna™: Target Patient Population
Table 21.21 Luxturna™: Net Present Value (USD Million)
Table 21.22 Luxturna™: Value Creation Analysis (USD Million)
Table 21.23 Zolgensma™: Target Patient Population
Table 21.24 Zolgensma™: Net Present Value (USD Million)
Table 21.25 Zolgensma™: Value Creation Analysis (USD Million)
Table 21.26 Collategene®: Target Patient Population
Table 21.27 Collategene®: Net Present Value (USD Million)
Table 21.28 Collategene®: Value Creation Analysis (USD Million)
Table 21.29 Libmedly™: Target Patient Population
Table 21.30 Libmedly™: Net Present Value (USD Million)
Table 21.31 Libmedly™: Value Creation Analysis (USD Million)
Table 21.32 Lumevaq® (GS010): Target Patient Population
Table 21.33 Lumevaq® (GS010): Net Present Value (USD Million)
Table 21.34 Lumevaq® (GS010): Value Creation Analysis (USD Million)
Table 21.35 OTL-103: Target Patient Population
Table 21.36 OTL-103: Net Present Value (USD Million)
Table 21.37 OTL-103: Value Creation Analysis (USD Million
Table 21.38 PTC-AADC: Target Patient Population
Table 21.39 PTC-AADC: Net Present Value (USD Million)
Table 21.40 PTC-AADC: Value Creation Analysis (USD Million)
Table 21.41 BMN 270: Target Patient Population
Table 21.42 BMN 270: Net Present Value (USD Million)
Table 21.43 BMN 270: Value Creation Analysis (USD Million)
Table 21.44 rAd-IFN/Syn3: Target Patient Population
Table 21.45 rAd-IFN/Syn3: Net Present Value (USD Million)
Table 21.46 rAd-IFN/Syn3: Value Creation Analysis (USD Million)
Table 21.47 beti-cel: Target Patient Population
Table 21.48 beti-cel: Net Present Value (USD Million)
Table 21.49 beti-cel: Value Creation Analysis (USD Million)
Table 21.50 eli-cel: Target Patient Population
Table 21.51 eli-cel: Net Present Value (USD Million)
Table 21.52 eli-cel: Value Creation Analysis (USD Million)
Table 21.53 lovo-cel: Target Patient Population
Table 21.54 lovo-cel: Net Present Value (USD Million)
Table 21.55 lovo-cel: Value Creation Analysis (USD Million)
Table 21.56 SRP-9001: Target Patient Population
Table 21.57 SRP-9001: Net Present Value (USD Million)
Table 21.58 SRP-9001: Value Creation Analysis (USD Million)
Table 21.59 EB-101: Target Patient Population
Table 21.60 EB-101: Net Present Value (USD Million)
Table 21.61 EB-101: Value Creation Analysis (USD Million)
Table 21.62 ProstAtak®: Target Patient Population
Table 21.63 ProstAtak®: Net Present Value (USD Million)
Table 21.64 ProstAtak®: Value Creation Analysis (USD Million)
Table 21.65 D-Fi: Target Patient Population
Table 21.66 D-Fi: Net Present Value (USD Million)
Table 21.67 D-Fi: Value Creation Analysis (USD Million)
Table 21.68 CG0070: Target Patient Population
Table 21.69 CG0070: Net Present Value (USD Million)
Table 21.70 CG0070: Value Creation Analysis (USD Million)
Table 21.71 Vigil™-EWS: Target Patient Population
Table 21.72 Vigil™-EWS: Net Present Value (USD Million)
Table 21.73 Vigil™-EWS: Value Creation Analysis (USD Million)
Table 21.74 Engensis®: Target Patient Population
Table 21.75 Engensis®: Net Present Value (USD Million)
Table 21.76 Engensis®: Value Creation Analysis (USD Million)
Table 21.77 VGX-3100: Target Patient Population
Table 21.78 VGX-3100: Net Present Value (USD Million)
Table 21.79 VGX-3100: Value Creation Analysis (USD Million)
Table 21.80 Invossa™ (TG-C): Target Patient Population
Table 21.81 Invossa™ (TG-C): Net Present Value (USD Million)
Table 21.82 Invossa™ (TG-C): Value Creation Analysis (USD Million)
Table 21.83 VYJUVEKT™: Target Patient Population
Table 21.84 VYJUVEKT™: Net Present Value (USD Million)
Table 21.85 VYJUVEKT™: Value Creation Analysis (USD Million)
Table 21.86 PF-06939926: Target Patient Population
Table 21.87 PF-06939926: Net Present Value (USD Million)
Table 21.88 PF-06939926: Value Creation Analysis (USD Million)
Table 21.89 PF-06838435: Target Patient Population
Table 21.90 PF-06838435: Net Present Value (USD Million)
Table 21.91 PF-06838435: Value Creation Analysis (USD Million)
Table 21.92 PF-07055480: Target Patient Population
Table 21.93 PF-07055480: Net Present Value (USD Million)
Table 21.94 PF-07055480: Value Creation Analysis (USD Million)
Table 21.95 SPK-8011: Target Patient Population
Table 21.96 SPK-8011: Net Present Value (USD Million)
Table 21.97 SPK-8011: Value Creation Analysis (USD Million)
Table 21.98 AMT-061: Target Patient Population
Table 21.99 AMT-061: Net Present Value (USD Million)
Table 21.100 AMT-061: Value Creation Analysis (USD Million)
Table 21.101 VB-111: Target Patient Population
Table 21.102 VB-111: Net Present Value (USD Million)
Table 21.103 VB-111: Value Creation Analysis (USD Million)
Table 21.104 Generx®: Target Patient Population
Table 21.105 Generx®: Net Present Value (USD Million)
Table 21.106 Generx®: Value Creation Analysis (USD Million)
Table 21.107 AMG001: Target Patient Population
Table 21.108 AMG001: Net Present Value (USD Million)
Table 21.109 AMG001: Value Creation Analysis (USD Million)
Table 21.110 OAV-101: Target Patient Population
Table 21.111 OAV-101: Net Present Value (USD Million)
Table 21.112 OAV-101: Value Creation Analysis (USD Million)
Table 21.113 ADXS-HPV: Target Patient Population
Table 21.114 ADXS-HPV: Net Present Value (USD Million)
Table 21.115 ADXS-HPV: Value Creation Analysis (USD Million)
Table 21.116 AGTC 501: Target Patient Population
Table 21.117 AGTC 501: Net Present Value (USD Million)
Table 21.118 AGTC 501: Value Creation Analysis (USD Million)
Table 21.119 LYS-SAF302: Target Patient Population
Table 21.120 LYS-SAF302: Net Present Value (USD Million)
Table 21.121 LYS-SAF302: Value Creation Analysis (USD Million)
Table 21.122 NFS-01: Target Patient Population
Table 21.123 NFS-01: Net Present Value (USD Million)
Table 21.124 NFS-01: Value Creation Analysis (USD Million)
Table 21.125 AG0302-COVID19: Target Patient Population
Table 21.126 AG0302-COVID19: Net Present Value (USD Million)
Table 21.127 AG0302-COVID19: Value Creation Analysis (USD Million)
Table 21.128 RGX-314: Target Patient Population
Table 21.129 RGX-314: Net Present Value (USD Million)
Table 21.130 RGX-314: Value Creation Analysis (USD Million)
Table 21.131 Hologene 5: Target Patient Population
Table 21.132 Hologene 5: Net Present Value (USD Million)
Table 21.133 Hologene 5: Value Creation Analysis (USD Million)
Table 22.1 Small-scale Cell Culture Systems
Table 22.2 List of Contract Manufacturing Service Providers for Viral Vectors and Plasmid DNA
Table 26.1 Gene Therapies: Distribution by Stage of Development
Table 26.2 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Phase of Development
Table 26.3 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Therapeutic Area
Table 26.4 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Phase of Development and Therapeutic Area
Table 26.5 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Vector Used
Table 26.6 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Gene / Molecule Targeted
Table 26.7 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Therapy
Table 26.8 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Vector Used and Type of Therapy
Table 26.9 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Gene Delivery Method Used
Table 26.10 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Route of Administration
Table 26.11 Early-Stage Pipeline of Gene Therapies: Distribution by Phase of Development
Table 26.12 Early-Stage Pipeline of Gene Therapies: Distribution by Therapeutic Area
Table 26.13 Early-Stage Pipeline of Gene Therapies: Distribution by Phase of Development and Therapeutic Area
Table 26.14 Early-Stage Pipeline of Gene Therapies: Distribution by Type of Vector Used
Table 26.15 Early-Stage Pipeline of Gene Therapies: Distribution by Type of Therapy
Table 26.16 Early-Stage Pipeline of Gene Therapies: Distribution by Type of Vector Used and Type of Therapy
Table 26.17 Early-Stage Pipeline of Gene Therapies: Distribution by Type of Gene Delivery Method Used
Table 26.18 Early-Stage Pipeline of Gene Therapies: Distribution by Route of Administration
Table 26.19 Gene Therapies: Distribution by Special Designation(s) Awarded
Table 26.20 Gene Therapy Developers: Distribution by Year of Establishment
Table 26.21 Gene Therapy Developers: Distribution by Company Size
Table 26.22 Gene Therapy Developers: Distribution by Location of Headquarters
Table 26.23 Gene Therapy Developers: Distribution by Year of Establishment, Company Size and Location of Headquarters
Table 26.24 Most Active Players: Distribution by Number of Gene Therapies
Table 26.25 Most Active Players: Distribution by Number of Gene Therapies and Phase of Development
Table 26.26 Gene Therapies: Distribution by Therapeutic Area and Special Designation(s) Awarded
Table 26.27 Gene Therapies for Oncological Diseases: Distribution by Target Indication and Phase of Development
Table 26.28 Gene Therapies for Oncological Diseases: Distribution by Type of Vector Used
Table 26.29 Gene Therapies for Neurological Diseases: Distribution by Target Indication and Phase of Development
Table 26.30 Gene Therapies for Neurological Diseases: Distribution by Type of Vector Used
Table 26.31 Gene Therapies for Ophthalmic Diseases: Distribution by Target Indication and Phase of Development
Table 26.32 Gene Therapies for Ophthalmic Diseases: Distribution by Type of Vector Used
Table 26.33 Gene Therapies for Metabolic Diseases: Distribution by Target Indication and Phase of Development
Table 26.34 Gene Therapies for Metabolic Diseases: Distribution by Type of Vector Used
Table 26.35 Gene Therapies for Genetic Diseases: Distribution by Target Indication and Phase of Development
Table 26.36 Gene Therapies for Genetic Diseases: Distribution by Type of Vector Used
Table 26.37 Gene Therapy Patent Portfolio: Distribution by Type of Patent
Table 26.38 Gene Therapy Patent Portfolio: Cumulative Distribution by Publication Year, 2017-2022
Table 26.39 Gene Therapy Patent Portfolio: Distribution by Type of Patent and Publication Year
Table 26.40 Gene Therapy Patent Portfolio: Distribution by Geography
Table 26.41 Gene Therapy Patent Portfolio: North America Scenario
Table 26.42 Gene Therapy Patent Portfolio: Europe Scenario
Table 26.43 Gene Therapy Patent Portfolio: Asia-Pacific Scenario
Table 26.44 Gene Therapy Patent Portfolio: Distribution by CPC Symbols
Table 26.45 Gene Therapy Patent Portfolio: Leading Industry Players: Distribution by Number of Patents
Table 26.46 Gene Therapy Patent Portfolio (Sanofi and Genethon): Benchmarking by Patent Characteristics
Table 26.47 Gene Editing Patent Portfolio: Distribution by Type of Patent
Table 26.48 Gene Editing Patent Portfolio: Cumulative Distribution by Publication Year, 2017-2022
Table 26.49 Gene Editing Patent Portfolio: Distribution by Type of Patent and Publication Year
Table 26.50 Gene Editing Patent Portfolio: Distribution by Geography
Table 26.51 Gene Editing Patent Portfolio: North America Scenario
Table 26.52 Gene Editing Patent Portfolio: Europe Scenario
Table 26.53 Gene Editing Patent Portfolio: Asia-Pacific Scenario
Table 26.54 Gene Editing Patent Portfolio: Distribution by CPC Symbols
Table 26.55 Gene Editing Patent Portfolio: Leading Industry Players: Distribution by Number of Patents
Table 26.56 Gene Editing Patent Portfolio (Intellia Therapeutics and CRISPR Therapeutics): Benchmarking by Patent Characteristics
Table 26.57 Mergers and Acquisitions: Cumulative Year-Wise Trend, 2015-2022
Table 26.58 Mergers and Acquisitions: Distribution by Type of Agreement
Table 26.59 Mergers and Acquisitions: Distribution by Year and Type of Agreement
Table 26.60 Mergers and Acquisitions: Intercontinental and Intracontinental Deals
Table 26.61 Mergers and Acquisitions: Local and International Deals
Table 26.62 Acquisitions: Distribution by Key Value Drivers
Table 26.63 Acquisitions: Distribution by Year of Acquisition and Key Value Drivers
Table 26.64 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company’s Product
Table 26.65 Mergers and Acquisitions: Distribution by Year of Acquisition and Phase of Development of the Acquired Company’s Product
Table 26.66 Mergers and Acquisitions: Distribution by Therapeutic Area
Table 26.67 Funding and Investments: Cumulative Distribution of Instances by Year, 2015-2022
Table 26.68 Funding and Investments: Cumulative Distribution of Amount Invested by Year, 2015-2022 (USD Million)
Table 26.69 Funding and Investments: Distribution of Instances by Type of Funding, 2015-2022
Table 26.70 Funding and Investments: Distribution of Total Amount Invested by Type of Funding, 2015-2022 (USD Million)
Table 26.71 Funding and Investments: Summary of Investments, 2015-2022 (USD Million)
Table 26.72 Funding and Investments: Distribution of Instances and Amount Invested by Type of Therapy
Table 26.73 Funding and Investments: Distribution of Instances and Amount Invested by Geography
Table 26.74 Funding and Investments: Regional Distribution of Funding Instances
Table 26.75 Funding and Investments: Distribution of Instances and Amount Invested by Highest Phase of Development of the Company’s Product
Table 26.76 Funding and Investments: Distribution of Instances by Therapeutic Area
Table 26.77 Most Active Players: Distribution by Number of Funding Instances, 2015-2022
Table 26.78 Most Active Players: Distribution by Amount Invested, 2015-2022 (USD Million)
Table 26.79 Funding and Investments: Distribution of Instances by Type of Investors
Table 26.80 Leading Investors: Distribution by Number of Instances
Table 26.81 Funding and Investment Summary, 2015-2022 (USD Million)
Table 26.82 Clinical Trial Analysis: Distribution by Trial Registration Year, 2017-2022
Table 26.83 Clinical Trial Analysis: Distribution by Trial Status
Table 26.84 Clinical Trial Analysis: Distribution by Trial Phase
Table 26.85 Clinical Trial Analysis: Distribution by Therapeutic Area
Table 26.86 Clinical Trial Analysis: Distribution by Geography
Table 26.87 Clinical Trial Analysis: Distribution by Trial Registration Year and Geography
Table 26.88 Clinical Trial Analysis: Distribution by Trial Status and Geography
Table 26.89 Clinical Trial Analysis: Distribution by Type of Sponsor
Table 26.90 Clinical Trial Analysis: Distribution by Prominent Treatment Sites
Table 26.91 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Registration Year, 2017-2022
Table 26.92 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status
Table 26.93 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Phase
Table 26.94 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Therapeutic Area
Table 26.95 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Location of Trial Site
Table 26.96 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status and Location of Trial Site
Table 26.97 Demand Analysis: Overall Demand for Gene Therapies
Table 26.98 Demand Analysis: Distribution by Type of Therapy, 2022, 2030 and 2035
Table 26.99 Demand Analysis: Distribution by Therapeutic Area, 2022, 2030 and 2035
Table 26.100 Demand Analysis: Distribution by Geography, 2022, 2030 and 2035
Table 26.101 Demand Analysis: Country-wise Distribution (Asia-Pacific), 2022, 2030 and 2035
Table 26.102 Global Gene Therapy Market, 2022-2035 (USD Billion)
Table 26.103 Gene Therapy Market: Distribution by Therapeutic Area, 2022, 2030 and 2035
Table 26.104 Gene Therapy Market: Distribution by Type of Vector Used, 2022, 2030 and 2035
Table 26.105 Gene Therapy Market: Distribution by Type of Therapy, 2022, 2030 and 2035
Table 26.106 Gene Therapy Market: Distribution by Type of Gene Delivery Method Used, 2022, 2030 and 2035
Table 26.107 Gene Therapy Market: Distribution by Route of Administration, 2022, 2030 and 2035
Table 26.108 Gene Therapy Market: Distribution by Geography, 2022, 2030 and 2035
Table 26.109 Gene Therapy Market: Country-wise Distribution (Asia-Pacific), 2022, 2030 and 2035
Table 26.110 Gendicine® Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.111 Oncorine® Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.112 Rexin-G® Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.113 Neovasculgen® Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.114 Strimvelis® Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.115 Imlygic® Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.116 Luxturna™ Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.117 Zolgensma™ Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.118 Collategene® Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.119 Libmeldy™ Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.120 Lumevaq® (GS010) Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.121 OTL-103 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.122 PTC-AADC Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.123 BMN 270 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.124 rAd-IFN/Syn3 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.125 beti-cel Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.126 eli-cel Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.127 lovo-cel Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.128 SRP-9001 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.129 EB-101 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.130 ProstAtak® Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.131 D-Fi Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.132 CG0070 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.133 Vigil™-EWS Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.134 Engensis® Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.135 VGX-3100 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.136 Invossa™ (TG-C) Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.137 VYJUVEKT™ Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.138 PF-06939926 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.139 PF-06838435 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.140 PF-07055480 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.141 SPK-8011 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.142 AMT-061 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.143 VB-111 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.144 Generx® Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.145 AMG001 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.146 OAV-101 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.147 ADXS-HPV Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.148 AGTC 501 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.149 LYS-SAF302 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.150 NFS-01 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.151 AG0302-COVID19 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.152 RGX-314 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.153 Hologene 5 Sales Forecast (Till 2035): Base Scenario (USD Million)

List Of Companies

The following companies and organizations have been mentioned in the report:

  1. 3SBio
  2. 4basebio
  3. 4BIO Capital
  4. 4D Molecular Therapeutics
  5. 5AM Ventures
  6. 683 Capital Management
  7. AAVantgarde Bio
  8. AavantiBio
  9. aaVective
  10. AaviGen
  11. AAVogen
  12. AbbVie
  13. Abby Grace Foundation
  14. Abeona Therapeutics
  15. Abingworth
  16. Access Biotechnology
  17. Adage Capital Management
  18. Adrenas Therapeutics
  19. Advaxis
  20. Adverum Biotechnologies
  21. Aevitas Therapeutics
  22. Affinia Therapeutics
  23. Agathos Biologics
  24. Agent Capital
  25. AGTC
  26. Aisling Capital
  27. AJU IB Investment
  28. Akouos
  29. Alaska Permanent Fund
  30. Alexandria Venture Investments
  31. Alia Therapeutics
  32. Allergan
  33. AllStripes
  34. Ally Bridge Group
  35. Almirall
  36. Alpha Cognition
  37. Alpha Holdings
  38. AlphaVax
  39. Altheia Science
  40. Altitude Life Science Ventures
  41. Alumni Ventures
  42. Amarna Therapeutics
  43. Ambulero
  44. American Gene Technologies™
  45. Amgen
  46. Amicus Therapeutics
  47. Amplo
  48. Anaeropharma Science
  49. Andelyn Biosciences
  50. Andreessen Horowitz
  51. AnGes
  52. Angionetics
  53. Anjarium Biosciences
  54. Anlong Biopharmaceutical
  55. Apellis Pharmaceuticals
  56. ApolloBio
  57. Apple Tree Partners
  58. Arbor Biotechnologies
  59. ARCH Overage Fund
  60. ARCH Venture Partners
  61. Arctic Light Venture Capital
  62. Arix Bioscience
  63. Arkin Bio Ventures
  64. ArrowMark Partners
  65. Arthrogen
  66. ARTIS Ventures
  67. Aruvant Sciences
  68. Asabys Partners
  69. ASC Therapeutics
  70. Ascidian Therapeutics
  71. Asia Alpha
  72. Asklepios BioPharmaceutical
  73. Aspa Therapeutics
  74. Aspire Capital Fund
  75. Astellas Gene Therapies
  76. Astellas Pharma
  77. AstraZeneca
  78. Atamyo Therapeutics
  79. Atlas Venture
  80. Atsena Therapeutics
  81. Auburn University
  82. Audition Therapeutics
  83. Auriga Partners
  84. Australian Taxation Office
  85. Avellino Lab USA
  86. AviadoBio
  87. Avidity Partners
  88. Avrion Therapeutics
  89. AVROBIO
  90. Axovia Therapeutics
  91. Baillie Gifford
  92. Bain Capital Life Sciences
  93. Banca Esperia
  94. Bayer
  95. Beam Therapeutics
  96. Belief BioMed
  97. Berkeley Catalyst Fund
  98. Bessemer Trust
  99. Bezos Expeditions
  100. Bill & Melinda Gates Foundation
  101. Biogen
  102. Biointaxis
  103. BioInvent International
  104. Biolocity
  105. BioMarin Pharmaceutical
  106. Bionic Sight
  107. Biotechnology Value Fund
  108. BioVec Pharma
  109. Blue Bear Ventures
  110. bluebird bio
  111. BlueWater Angels
  112. Boehringer Ingelheim
  113. Bonus BioGroup
  114. Borea Therapeutics
  115. Boxer Capital
  116. Bpifrance
  117. Brace Pharma Capital
  118. Breton Capital
  119. BridgeBio
  120. BrightGene Bio-Medical Technology
  121. Bristol Myers Squibb
  122. Brookside Capital
  123. BVF Partners
  124. C4 Holding
  125. California Institute for Regenerative Medicine (CIRM)
  126. CAM Capital
  127. Cambridge Enterprise
  128. Cambridge Gene Therapy
  129. Cambridge Innovation Capital
  130. Camford Capital
  131. Canada Pension Plan Investment Board
  132. CANbridge Pharmaceuticals
  133. Cancer Research Institute
  134. Cancer Research UK
  135. Candel Therapeutics
  136. CanSino Biologics
  137. CapDecisif Management
  138. Capsida Biotherapeutics
  139. Carmine Therapeutics
  140. Casdin Capital
  141. Case Western Reserve University
  142. Castle Creek Biosciences
  143. Catalyst Biosciences
  144. CavoGene LifeSciences
  145. CCRM Enterprises
  146. CDG Capital
  147. Celgene
  148. CellGenTech
  149. Celsion
  150. Cenova Capital
  151. CG Oncology
  152. CGP Investment
  153. Charcot–Marie–Tooth Association
  154. Chende Capital
  155. Chiba University
  156. Chiesi Ventures
  157. Children’s Medical Research Foundation
  158. Children’s Oncology Group
  159. CICC
  160. Cima Universidad de Navarra
  161. CincyTech
  162. CITIC Group
  163. CITIC Medical Fund
  164. Clarus Ventures
  165. Clearside Biomedical
  166. CLINO
  167. Clough Capital Partners
  168. CMG-SDIC Capital
  169. Coalition for Epidemic Preparedness Innovations
  170. Coave Therapeutics
  171. CODA Biotherapeutics
  172. Code Biotherapeutics
  173. Codexis
  174. Columbia University
  175. Columbus Venture Partners
  176. CombiGene
  177. Conectus
  178. Consort Medical
  179. Copernicus Therapeutics
  180. Cormorant Asset Management
  181. Cowen
  182. Cowen Healthcare Investments
  183. Cowen Private Investments
  184. CRISPR Therapeutics
  185. CSL Behring
  186. CureDuchenne Ventures
  187. Cynvec
  188. Cyprium Therapeutics
  189. Cystic Fibrosis Foundation
  190. D1 Capital Partners
  191. Daiichi Sankyo
  192. DARPA
  193. Decheng Capital
  194. Decibel Therapeutics
  195. Deerfield Management
  196. Delphi Genetics  
  197. Dexcel Pharma
  198. DH-LT-Investments
  199. DiNAQOR
  200. DNAlite Therapeutics
  201. DNAtrix
  202. Driehaus Capital Management
  203. Duke University
  204. Dyno Therapeutics
  205. EB Medical Research Foundation
  206. EB Research Partnership (EBRP)
  207. EcoR1 Capital
  208. EDBI
  209. Editas Medicine
  210. Edmond de Rothschild Investment Partners
  211. Eight Roads Ventures
  212. Eikonoklastes Therapeutics
  213. Elaia
  214. ElevateBio
  215. Eli Lilly
  216. Emendo Biotherapeutics
  217. Emory University
  218. Encoded Therapeutics
  219. Endsulin
  220. enGene
  221. Ennovation Ventures
  222. Enochian BioSciences
  223. Ensoma
  224. Entos Pharmaceuticals
  225. Epeius Biotechnologies
  226. EPIQ Capital Group
  227. EPS Holdings
  228. Esco Ventures
  229. Esteve
  230. Emerging Technology Partners (ETP)
  231. Eureka Eurostars
  232. EUROFANCOLEN
  233. European Innovation Council Accelerator
  234. European Investment Bank
  235. Eventide Asset Management
  236. Evox Therapeutics
  237. EVX Ventures
  238. Excision BioTherapeutics
  239. Exegenesis Bio
  240. Exen Capital
  241. Expression Therapeutics
  242. ExSight Capital
  243. Eyevensys
  244. F2 Ventures
  245. Farallon Capital Management
  246. FAUN Foundation
  247. FEDER
  248. FerGene
  249. Fidelity Biosciences
  250. Fidelity Management & Research Company
  251. Fidim
  252. Fin Posillipo
  253. Flagship Pioneering
  254. Flash Therapeutics
  255. Flerie Invest
  256. Flexion Therapeutics
  257. Fondation Sanfilippo Suisse
  258. Forbion
  259. Foresite Capital
  260. Forge Biologics
  261. Fosun Capital
  262. Foundation Fighting Blindness
  263. Fountain Healthcare Partners
  264. FoxKiser
  265. F-Prime Capital
  266. Franklin Templeton Investments
  267. Frazier Healthcare Partners
  268. Freeline Therapeutics
  269. Friedreich’s Ataxia Research Alliance
  270. Fund+
  271. Fundación Sanfilippo B
  272. G&P Biosciences
  273. Galia Gestion
  274. Gene Biotherapeutics
  275. Gene Therapy Research Institution
  276. GeneCure Biotechnologies
  277. Genelux
  278. GeneMedicine
  279. Genenta Science
  280. GeneOne Life Science
  281. GeneQuine Biotherapeutics
  282. Generation Bio
  283. Genespire
  284. Genethon
  285. Genexine
  286. Genflow Biosciences
  287. Genprex
  288. GenSight Biologics
  289. Genzyme
  290. Georgia Research Alliance
  291. Ghost Tree Capital Group
  292. Gimv
  293. GO CAPITAL
  294. Go Capital Amorçage
  295. GOFAR
  296. GOG Foundation
  297. Google Ventures
  298. GordonMD Global Investments
  299. Gradalis
  300. Graphite Bio
  301. GreatPoint Ventures
  302. Green Cross Holdings
  303. Guofang Capital
  304. Gyroscope Therapeutics
  305. Harvard University
  306. Hatteras Venture Partners
  307. Hayfin Capital Management
  308. HBM Healthcare Investments
  309. HealthCap
  310. Helixmith
  311. Hemera Biosciences
  312. Herantis Pharma
  313. Hillhouse Capital Group
  314. Hiroshima Venture Capital
  315. HIV Vaccine Trials Network
  316. Holostem Terapie Avanzate
  317. Homology Medicines
  318. Horizon Technology Finance
  319. Hovione Scientia
  320. Huapont Life Sciences
  321. Huida (Shanghai) Biotechnology
  322. Human Stem Cells Institute PJSC (HSCI)
  323. Idinvest Partners
  324. iECURE
  325. Ikarian Capital
  326. Ikarovec
  327. Illumina Ventures
  328. Ilya Pharma
  329. Imperial Innovations
  330. Imperial Innovations Group
  331. Imvus
  332. Inception Capital Management
  333. New Energy and Industrial Technology Development Organization (NEDO)
  334. InnoSkel
  335. Innovate UK
  336. Innovation Angel Funds
  337. Innovation Network Corporation of Japan
  338. Innovation Norway
  339. InnoVision Capital
  340. Inovio Pharmaceuticals
  341. Inserm Transfert
  342. Institute of Myology
  343. Intas Pharmaceuticals
  344. Intellia Therapeutics
  345. InverVest
  346. Investitionsbank des Landes Brandenburg
  347. Invus
  348. Israel Innovation Authority
  349. IVERIC bio
  350. Jaguar Gene Therapy
  351. Janssen Pharmaceuticals
  352. Janus Capital Management
  353. Janus Henderson Investors
  354. Jasper Therapeutics
  355. Jeito Capital
  356. Jennison Associates
  357. Jeune
  358. Jinwei
  359. Johnson & Johnson Development
  360. Juda Capital
  361. K2 Venture Capital
  362. Kate Therapeutics
  363. Kendall Capital Partners
  364. Kepler Cheuvreux
  365. Keytone Ventures
  366. Kissei Pharmaceutical
  367. Kiwoom Securities
  368. Kodikaz Therapeutic Solutions
  369. Kohlberg Kravis Roberts (KKR)
  370. Kolon TissueGene
  371. Korea Investment Partners
  372. Kriya Therapeutics
  373. Krystal Biotech
  374. Kubota Vision
  375. Kurma Partners
  376. Kyorin Pharmaceutical
  377. Lacerta Therapeutics
  378. Leerink Partners
  379. Lee's Pharmaceutical
  380. Legend Capital
  381. Leiden University Medical Center
  382. Leland Stanford Junior University
  383. Lentistem® Biotech
  384. Centre Léon Bérard
  385. Lepu Medical Technology
  386. LEXEO Therapeutics
  387. Life Sciences Partners (LSP)
  388. LifeArc
  389. Lightstone Ventures
  390. Lilly Asia Ventures
  391. LIME Asset Management
  392. Lipigon Pharmaceuticals
  393. LogicBio Therapeutics
  394. Lokon Pharma
  395. Longevity Biotech
  396. Longitude Capital
  397. Longmen Capital
  398. Longwood Fund
  399. LP Investments
  400. Ludwig Cancer Research
  401. Lumira Ventures
  402. Lundbeckfond Ventures
  403. LYFE Capital
  404. Lysogene
  405. Malin
  406. Mangrove Partners
  407. Maryland Technology Development
  408. Matrix Capital Management
  409. Matrix Partners China
  410. Maverick Ventures
  411. MDimune
  412. Mediphage Bioceuticals
  413. Medison Ventures
  414. MeiraGTx
  415. Memorial Sloan Kettering Cancer Center
  416. Mercia Technologies
  417. Merck
  418. MERITZ Securities
  419. Metagenomi
  420. Michigan Economic Development
  421. MidCap Financial
  422. Milo Biotechnology
  423. Mirae Asset Global Investments
  424. Mirum Pharmaceuticals
  425. Mita Securities
  426. Mitsubishi Tanabe Pharma
  427. Mitsubishi UFJ Capital
  428. Moderna
  429. Moffitt Cancer Center
  430. Momotaro-Gene
  431. Monograph Capital
  432. Morningside
  433. Movember
  434. MPM Capital
  435. MultiVir
  436. Muscular Dystrophy Association
  437. Mustang Bio
  438. Myonexus Therapeutics
  439. Myosana Therapeutics
  440. Myrtelle
  441. NanoCor Therapeutics
  442. Nanoscope Therapeutics
  443. Narya Capital
  444. National Cancer Institute
  445. National Eye Institute
  446. National Institute of Allergy and Infectious Diseases
  447. National Institute of Child Health and Human Development
  448. National Institute of Neurological Disorders and Stroke
  449. National Institutes of Health
  450. National MPS Society
  451. Nationwide Children’s Hospital
  452. Navega Therapeutics
  453. National Education Association (NEA)
  454. NEC
  455. Neo Gene Pharm
  456. NeoMed
  457. Nerveda
  458. Netherlands Enterprise Agency
  459. NeuExcell Therapeutics
  460. Neuracle Genetics
  461. Neurocrine Biosciences
  462. Neurogene
  463. Neurophth Therapeutics
  464. Neuvocor
  465. New Enterprise Associates
  466. New Leaf Venture Partners
  467. Newsoara BioPharma
  468. Ninevah Therapeutics
  469. Nissei Capital
  470. Noga Therapeutics
  471. Nolan Capital
  472. Bioscience Innovation Grant Program (BIG)
  473. Northern Light Venture Capital
  474. Northpond Ventures
  475. Novartis
  476. Novo Holdings
  477. Novo Nordisk
  478. Nuntius Therapeutics
  479. Oberland Capital
  480. Obsidian Therapeutics
  481. Ocugen
  482. Odylia Therapeutics
  483. Oisin Biotechnologies
  484. Omega Funds
  485. Omnes Capital
  486. Oncolys BioPharma
  487. Oncorus
  488. OncoSec Immunotherapies
  489. OncoSenX
  490. Opus Genetics
  491. OrbiMed
  492. ORCA Therapeutics
  493. Orchard Therapeutics
  494. ORI Capital
  495. Orphinic Scientific
  496. Osage University Partners
  497. Osaka University
  498. OSI Investment Management
  499. Otonomy
  500. Oxford BioMedica
  501. Oxford Finance
  502. Oxular
  503. Pacira BioSciences
  504. Panmure Gordon
  505. Pappas Capital
  506. Paragon Biosciences
  507. Parker Institute for Cancer Immunotherapy
  508. Parkwalk
  509. Partner Fund Management
  510. Partners Innovation Fund
  511. Partners Investment
  512. Passage Bio
  513. Patient Square Capital
  514. Pattern BioSciences
  515. Pavilion Capital
  516. PBM Capital
  517. Pentwater Capital Management
  518. Perceptive Advisors
  519. PeriphaGen
  520. Perseverance Capital Management
  521. Pfizer
  522. PFM Health Sciences
  523. Pharmakon Advisors
  524. Pharming Group
  525. Phoenix Nest
  526. PhorMed
  527. Pivotal bioVenture Partners
  528. PlasmidFactory
  529. Pontifax
  530. POSCO Capital
  531. Poseida Therapeutics
  532. Precigen
  533. Precision BioSciences
  534. Prostate Cancer Foundation
  535. PsiOxus Therapeutics
  536. PTC Therapeutics
  537. Purespring Therapeutics
  538. Qianhai Funds of Funds
  539. Qianzhan Investment Management
  540. Qiming Venture Partners
  541. QVT Financial
  542. RA Capital Management
  543. RBV Capital
  544. RD Fund
  545. Red Sanfilippo Foundation
  546. Redbiotec
  547. Redmile Group
  548. Redpin Therapeutics
  549. Reflection Biotechnologies
  550. Regeneron Pharmaceuticals
  551. REGENXBIO
  552. Rejuvenate Bio
  553. Remedium Bio
  554. Renova Therapeutics
  555. Research Council of Norway
  556. Rev1 Ventures
  557. Reyon Pharmaceutical
  558. Ridgeback Capital Investments
  559. Ring Therapeutics
  560. Roche
  561. Rock Springs Capital Management
  562. Rocket Pharmaceuticals
  563. Roswell Park Comprehensive Cancer Center
  564. Royal Holloway University of London
  565. Royal Melbourne Hospital
  566. RTW Investments
  567. Saifu Pharmaceutical
  568. SalioGen Therapeutics
  569. Samsara BioCapital
  570. Samum Vermögensverwaltungs
  571. San Rocco Therapeutics
  572. Sana Biotechnology
  573. Sanfilippo Children’s Foundation
  574. Sangamo Therapeutics
  575. Sanofi Genzyme
  576. Santen Pharmaceutical
  577. Sarepta Therapeutics
  578. SBI Japan-Israel Innovation Fund
  579. Scancell
  580. Schroder Adveq
  581. Scottish Enterprise
  582. S-Cubed Capital
  583. SDL Ventures
  584. Seattle Children’s Research Institute
  585. Sectoral Asset Management
  586. Selecta Biosciences
  587. Seneca Therapeutics
  588. Sensorion
  589. Sequoia Capital China
  590. Seroba Life Sciences
  591. SAFE Pharmaceutical
  592. Seven and Eight Biopharmaceuticals
  593. Seventure Partners
  594. Sham Innovation Santé
  595. Shanghai BDgene Technology
  596. Shanghai Sunway Biotech
  597. Shape Therapeutics
  598. Shenzhen Qianhai Taxus
  599. Sherpa Healthcare Partners
  600. Shinhan Capital
  601. Shinsei Corporate Investment
  602. Sibiono GeneTech
  603. Sight Again
  604. Silicon Valley Bank
  605. SillaJen
  606. Sio Gene Therapies
  607. Sirtex Medical
  608. SiSaf
  609. Skyline Therapeutics
  610. SMBC Venture Capital
  611. Sofinnova Ventures
  612. Solid Biosciences
  613. Song Hong Fang
  614. Sonova
  615. Sorrento Therapeutics
  616. SOSV
  617. Spanish Ministry of Science
  618. SparingVision
  619. Spark Therapeutics
  620. Sphera Global Healthcare Fund
  621. Spirovant Sciences
  622. SpliceBio
  623. SR One
  624. SR-Tiget
  625. St. Jude Children’s Research Hospital
  626. Stockholm University
  627. Stop Sanfilippo Fundación
  628. StrideBio
  629. STRM.BIO
  630. Surveyor Capital
  631. Suvretta Capital Management
  632. SV Health Investors
  633. SVB Leerink
  634. SVE Capital
  635. SwanBio Therapeutics
  636. Swedbank Robur
  637. Syncona
  638. T. Rowe Price Associates
  639. Takeda Pharmaceutical
  640. Tamid Bio
  641. Target ALS Foundation
  642. Targovax
  643. Taysha Gene Therapies
  644. Team Sanfilippo Foundation
  645. TeamedOn International
  646. Tech Coast Angels
  647. TechLife Capital
  648. Temasek
  649. Temple University
  650. Tenaya Therapeutics
  651. Tetraneuron
  652. The Column Group (TCG)
  653. The Invus Group
  654. Théa Open Innovation
  655. TheraBiologics
  656. Theravectys
  657. Third Rock Ventures
  658. ToolGen
  659. TPG Capital
  660. Transgene
  661. Transhuman Capital
  662. Trentino Invest
  663. Triumph Venture Capital
  664. Trizell
  665. Tsinghua Holdings Capital
  666. Tufts University
  667. Turenne
  668. Tyris Therapeutics
  669. UCB
  670. UCL Technology Fund
  671. UK Cystic Fibrosis Gene Therapy Consortium
  672. UK Innovation & Science Seed Fund
  673. Ultragenyx Pharmaceutical
  674. UMass Chan Medical School
  675. UNC Gene Therapy Center
  676. uniQure Biopharma
  677. Universitat Autònoma de Barcelona (UAB)
  678. University College London
  679. University Hospitals Cleveland Medical Center
  680. University of Alabama at Birmingham
  681. University of California, Berkeley
  682. University of California San Diego School of Medicine
  683. University of California San Francisco
  684. University of Florida
  685. University of Leeds
  686. University of Massachusetts
  687. University of Massachusetts Boston
  688. University of Minnesota
  689. University of Modena and Reggio Emilia
  690. University of North Carolina at Chapel Hill
  691. University of Padua
  692. University of Pennsylvania
  693. University of Pittsburgh
  694. University of Washington
  695. University of Washington School of Medicine
  696. UPMC Enterprises
  697. Uppsala University
  698. Urovant Sciences
  699. Usher 2020 Foundation
  700. Valencian Innovation Agency
  701. Valencian Institute of Finance
  702. Valor Equity Partners
  703. V-Bio Ventures
  704. VBL Therapeutics
  705. VCN Biosciences
  706. VectorY Therapeutics
  707. venBio
  708. Venrock
  709. Versant Ventures
  710. Vertex Pharmaceuticals
  711. Vickers Venture Partners
  712. Vida Ventures
  713. Vigene Biosciences
  714. ViGeneron
  715. Viking Global Investors
  716. ViralGEN
  717. Vivet Therapeutics
  718. Vivo Capital
  719. Voyager Therapeutics
  720. VREX Therapeutics
  721. Washington Research Foundation
  722. Washington State University
  723. Waverly Capital
  724. Weill Cornell Medicine
  725. Weizmann Institute of Science
  726. Wellcome Trust
  727. Wellington Management
  728. Westlake Village BioPartners
  729. Whitesun Healthcare Ventures
  730. WI Harper
  731. Wize Pharma
  732. Woodford Investment Management
  733. Woodline Partners
  734. Wu Capital
  735. Wuhan Binhui Biotechnology
  736. WuXi Advanced Therapies
  737. WuXi AppTec
  738. Wyvern Pharmaceuticals
  739. Xalud Therapeutics
  740. XyloCor Therapeutics
  741. Yangtze River Pharmaceutical
  742. Ysios Capital
  743. Yuhan
  744. Ziopharm Oncology

Source: www.globenewswire.com/news-release/2020/12/21/2148486/0/en/Orchard-Therapeutics-Receives-EC-Approval-for-Libmeldy-for-the-Treatment-of-Early-Onset-Metachromatic-Leukodystrophy-MLD.html

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