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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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.

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In August 2017, Tisagenlecleucel became the first FDA approved gene therapy for the treatment of relapsed or refractory pediatric and young-adult B-cell acute lymphoblastic leukemia. 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 companies 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 gene therapy 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 gene therapy companies and the growing demand for a single dose of effective therapeutic, the gene therapy market is anticipated to witness significant market growth during the forecast period.

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 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

Key Drugs in Gene Therapy Market  

Examples of key drugs in gene therapy market that are phase II or above of clinical deevopment (which have also been profiled in this market research report; the complete list of drugs and gene therapy companies 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. Further, some examples of fda approved gene therapy include (in alphabetical order) ABECMA (Celgene Corporation), ADSTILADRIN (Ferring Pharmaceuticals), ELEVIDYS (Sarepta Therapeutics), HEMGENIX (CSL Behring), IMLYGIC (BioVex), PROVENGE (Dendreon Corporation) and ZYNTEGLO (bluebird bio)
This market research report includes an easily searchable excel database of all the gene therapy companies and their products, worldwide.

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.
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 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 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

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 market report features an extensive study of the current market landscape, market size, market forecast 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 gene therapy companies 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 gene therapy companies.
  • 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.
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

A discussion on the various operating models adopted by gene therapy companies 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 this market report is to provide a detailed market forecast analysis in order to estimate the existing market size and future opportunity associated with gene therapies during the forecast period. Based on multiple parameters, likely adoption trends and through primary validations, we have provided an informed estimate on the market evolution during the forecast 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

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.

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