Top Gene Therapy Companies: The Innovators Making a Difference

Published: August 2024

The gene therapy market is witnessing robust growth, propelled by several key drivers, including increasing regulatory approvals, expanding clinical research, technological advancements, growing demand, and rising investments in this sector.We analyzed over 240 gene therapy companies that are engaged in development of gene therapy for various indications. In this article, discover eight top gene therapy companies and their capabilities in the overall gene therapy market.

Health disorders arising from congenital abnormalities and inherited genetic mutations have a profound impact on individuals throughout their lives. These chronic conditions encompass a diverse range of disorders, including autoimmune disorders, genetic disorders, neurological disorders and oncological disorders. Such disorders arise from inherited genetic anomalies that disrupt the normal functioning of genes and adversely affect the protein encoding process. According to the World Health Organization (WHO), 10 out of every 1,000 individuals are affected by genetic disorders, with over 70 million people worldwide living with such conditions. In fact, more than 40% of infant mortality globally is associated with various genetic disorders. Therefore, in order to address the aforementioned challenges, several research initiatives are underway to develop effective methods for delivering targeted therapies directly to affected organs or cells. In this regard, gene therapies have emerged as a revolutionary approach and have proven to be a promising modality in treating wide array of diseases.

With the rapidly evolving gene therapy product portfolio, an increasing number of startups and big pharma players are entering the gene therapy market. Presently, 345 gene therapy companies are engaged in developing various early and late-stage therapies worldwide. Further, in recent years, there has been a significant increase in the integration of novel technologies, such as gene modification, genome editing, genome sequencing and manipulation technologies (molecular / gene switch), in conjugation with advanced gene delivery methods. Moreover, next-generation delivery platforms, including nanoparticles and hybrid vector systems, have demonstrated the capability to facilitate effective, precise and safe delivery of gene-based therapeutics, thereby enabling more targeted treatment approaches. Driven by the collective efforts of gene therapy companies coupled with advancements in technology and growing demand for single-dose effective therapeutics, the gene therapy market is poised to witness substantial market growth during the forecast period.

Roots Analysis has conducted an exhaustive study on Gene Therapy Market featuring the current market landscape and future opportunity for the companies engaged in developing gene therapies, over a span of 11 years.

Table of Contents

Below, we have listed (in alphabetical order), the top eight gene therapy companies:

Amgen
BioMarin Pharmaceutical
Bluebird bio
CRISPR Therapeutics
CSL Behring
Ferring Pharmaceuticals
Novartis
Sibiono GenTech

Top 8 Gene Therapy Companies

Interested in exploring all 240+ gene therapy companies and their recent initiatives?

Discover Below the Details on 8 Top Gene Therapy Companies

Let us deep dive to understand the activity of the 8 top gene therapy companies offering therapies for various indications. It is essential to note that the selection of top players can differ substantially according to the defined criteria.

Amgen Acquired Horizon Therapeutics for USD 27.8 billion, Enhancing its Capabilities in Developing Therapies for Severe Inflammatory Diseases

1. Amgen

Amgen is a biotechnology company, primarily focused on the discovery, development, manufacturing and delivery of novel human therapeutics that have the potential to profoundly impact patients' lives worldwide. The company claims to be engaged in the development of innovative and differentiated medicines that not only offer treatment benefits but also help in reducing the global burden of diseases.

Amgen: Company Overview

Particulars	Specifications
Company Logo
Year of Establisment	1980
Number of Employees	10,001+
Headquarters	US

The therapeutics developed by the firm are primarily focused on cardiovascular disorders, oncological disorders, bone health, neuroscience, nephrology and inflammation. Further, the company utilizes advanced human genetics to develop oncolytic immunotherapy viruses to cater to the unmet needs of addressing genetic mutations associated with various oncological indications. It is worth highlighting that the firm’s first gene therapy, IMLYGIC (an oncolytic immunotherapy virus) received the USFDA approval in 2015 for patients diagnosed with melanoma. Further, the company has launched Amgen Support Plus, a reimbursement scheme which provides financial assistance to patients, thereby increasing access to its various therapeutic products (including IMLYGIC) to a larger patient population.

Recent Developments:

In May 2023, Amgen entered into a collaboration with TScan Therapeutics with an aim to utilize TScan's proprietary target discovery platform to identify novel therapeutic targets for Crohn's disease.

In January 2022, Amgen announced a collaboration with Generate Biomedicines aimed at discovering and creating protein therapeutics. This partnership leverages Generate's machine learning-enabled technology platform, which is designed to enhance drug discovery processes, including those applicable to gene therapies.

Further details on recent initiatives taken by Amgen and other gene therapy companies have been provided in detail in the report.

BioMarin Pharmaceutical Collaborated with German National Association of Statutory Health Insurance Funds on the Reimbursement for ROCTAVIAN®

2. BioMarin Pharmaceutical

BioMarin Pharmaceutical is a global biotechnology company engaged in the development and commercialization of transformative medicine for various rare and genetic disorders. Leveraging its expertise in genomics, the firm has developed a robust pipeline of commercial, clinical and preclinical small molecules and biologics that are designed to directly address the fundamental cause of a disease.

BioMarin Pharmaceuticals: Company Details

Particulars	Specifications
Company Logo
Year of Establisment	1997
Number of Employees	1,001-5,000
Headquarters	US

The firm has developed two AAV gene therapies for the treatment of cardiovascular and hematological disorders. Additionally, the company has inked several partnerships with academic institutions and other biotechnology firms in order to advance its research and development efforts, ultimately striving to improve the lives of patients with unmet medical needs.

The company has signed an agreement with German National Association of Statutory Health Insurance Funds (GKV-SV) regarding the reimbursement for Roctavian (valoctocogene roxaparvovec-rvox), a gene therapy for severe hemophilia A. The price is set at EUR 28,933.53 per vial, which translates to approximately USD 900,000 in net revenue per patient, considering the average patient weight and the number of vials required for treatment, as well as customary discounts and rebates priced.

Recent Developments:

In June 2024, BioMarin presented new four-year data from its Phase 3 study at the International Society on Thrombosis and Haemostasis (ISTH) Congress. This data highlights the long-term safety and efficacy of ROCTAVIAN, showing durable bleed control and sustained expression of factor VIII four years post-infusion.

In August 2024, BioMarin announced a strategic shift regarding ROCTAVIAN, opting to concentrate its efforts on three key markets: the United States, Germany, and Italy. This decision includes downsizing its broader development activities while continuing to gather long-term data from existing patients.

Further details on recent initiatives taken by BioMarin Pharmaceuticals and other gene therapy companies have been provided in detail in the report.

FDA Announced the Approval of bluebird bio's Gene Therapy Lyfgenia (lovotibeglogene autotemcel) for Sickle Cell Disease

3. bluebird bio
bluebird bio is a biopharmaceutical company primarily focused on the discovery and development of gene therapies for various genetic disorders. The company leverages its proprietary HSC LVV platform technology and megaTAL technology to develop breakthrough ex-vivo gene therapies. Till date, the company has successfully commercialized three gene therapies for indications across genetic, hematological and neurological disorders.

bluebird bio: Company Overview

Particulars	Specifications
Company Logo
Year of Establisment	1993
Number of Employees	201-500
Headquarters	US

The firm has a robust pipeline of preclinical and discovery stage gene therapies that has the potential to either add therapeutic genes or suppress disease-causing genes, which is usually not possible with a conventional small molecule drug. Further, it is worth highlighting that the company has inked various reimbursement agreements with several medical insurance providers in order to ease affordability and enhance patient accessibility to it gene therapies.

Recent Developments:

In May 2024, bluebird bio announced the completion of the first commercial cell collection for Lyfgenia at a Qualified Treatment Centre. Creating the milestone in making the newly approved gene therapy available to patients with sickle cell disease in the real-world setting.

Further details on recent initiatives taken by bluebird bio and other gene therapy companies have been provided in detail in the report.

FDA Announced the Approval of Casgevy, a CRISPR-based Gene Editing Therapy Developed by CRISPR Therapeutics in Partnership with Vertex Pharmaceuticals in Order to Treat Sickle Cell Disease

4. CRISPR Therapeutics

CRISPR Therapeutics claims to be one of the leading gene editing therapy company focused on the development of transformative medicines using its proprietary CRISPR / Cas9 gene editing platform. This platform modifies, deletes, or corrects disease-causing abnormalities at its genetic sources.

CRISPR Therapeutics: Company Overview

Particulars	Specifications
Company Logo
Year of Establisment	2013
Number of Employees	201-500
Headquarters	US

With the help of CRISPR / Cas9 platform, the company offers a diverse pipeline of gene editing therapies with a primary focus on therapeutic areas, such as autoimmune, cardiovascular, oncological and rare disorders. It is worth highlighting that the company has collaborated with leading pharmaceutical companies in order to accelerate research and development of gene therapies for a myriad of conditions.

Recent Developments:

In February 2024, CRISPR Therapeutics announced a registered direct offering, raising USD 280 million. The funding is intended to support the expansion of their clinical pipeline and ongoing research efforts.

In March 2024, CRISPR Therapeutics reported a strong financial position with USD 2.1 billion in cash and marketable securities, which positions the company well for future investments and developments in gene therapy.

Further details on recent initiatives taken by CRISPR Therapeutics and other gene therapy companies have been provided in detail in the report.

CSL Behring Announced That the First Patient had Received its FDA-approved Gene Therapy HEMGENIX® in the United States in June 2023

5. CSL Behring

CSL Behring, a subsidiary of CSL, is a leading biopharmaceutical company involved in the development of innovative therapies for rare and serious diseases. The firm specializes in developing and manufacturing plasma-derived and recombinant therapeutic products.

CSL Behring: Company Overview

Particulars	Specifications
Company Logo
Year of Establisment	1997
Number of Employees	10,000+
Headquarters	US

The company operates across four segments, namely immunology, hematology, respiratory, and albumin. Within the hematology segment, the company has a vast portfolio of gene therapies for hemophilia B and other inherited hematological conditions. The company also provides products for the prevention of hemolytic disease of the newborns (HDN), infection in solid organ transplant recipients and to treat specific infections for victims of trauma and burns. Additionally, by leveraging the advanced genetic technologies, the firm aims to offer long-term, potentially curative treatments that shall transform the current standard of care achieved with conventional medicines. Further, the firm has inked several strategic collaborations and has made massive investments in cutting-edge research to further enhance the company’s capabilities in gene therapy development.

Recent Developments:

In March 2024, CSL Behring signed the first commercial agreement in Austria to fund HEMGENIX® (etranacogene dezaparvovec), the first gene therapy approved in Austria for the treatment of severe and moderately severe hemophilia B in adult patients.

In December 2023, CSL Behring was granted Direct Access for HEMGENIX® by the French Ministry of Health.

Further details on recent initiatives taken by CSL Behring and other gene therapy companies have been provided in detail in the report.

Ferring Pharmaceuticals Announced Capacity Expansion for Adstiladrin at its Facilities in Finland and the US; Capacity Expansion Projects to Reach Full Capacity by 2025

6. Ferring Pharmaceuticals

Ferring Pharmaceuticals is a global biopharmaceutical company committed to developing innovative treatments capable of improving the patient’s quality of life. The firm operates across various segments, including reproductive health, maternal health, gastroenterology, and urology.

Ferring Pharmaceuticals: Company Overview

Particulars	Specifications
Company Logo
Year of Establisment	1950
Number of Employees	5000-10,001
Headquarters	Switzerland

The company primarily focuses on harnessing the power of genetic science to address the underlying causes of chronic inherited conditions, particularly in areas wherein the conventional treatments have limited efficacy. Additionally, it is worth highlighting that the company’s gene therapy product Adstiladrin is the first and the only approved gene therapy for adults with non-muscle invasive bladder cancer.

Recent Developments:

In April 2024, Ferring Pharmaceuticals entered into a collaboration with SK pharmteco today announced an agreement to scale up commercial manufacturing capacity for the drug substance of Ferring’s intravesical gene therapy Adstiladrin® (nadofaragene firadenovec-vncg) for ensuring long-term future supply.

Further details on recent initiatives taken by Ferring and other gene therapy companies have been provided in detail in the report.

Novartis Continues to Evaluate Zolgensma Across a Robust Clinical Development Program and it is Now Approved in 47 Countries; More than 3,000 Patients Have Been Treated with the Therapy Globally

7. Novartis

Novartis is a healthcare company focused on discovery, development and manufacturing of generic pharmaceutical and eye care products. The company offers a wide range of drugs with the primary focus on various therapeutic areas such as oncological disorders, cardiovascular disorders, dermatological disorders, neurological disorders and various other disorders.

Novartis: Company Overview

Particulars	Specifications
Company Logo
Year of Establisment	1996
Number of Employees	10,000+
Headquarters	Switzerland

The firm leverages innovative technologies and strategies to produce high-value medicines that have consistently improved the patient’s quality of life over the past two decades. Additionally, by utilizing cutting-edge AAV vector technologies, the firm has built a robust gene therapy pipeline of more than 20 therapies intended for the treatment of life-threatening diseases. It is worth highlighting that the company’s gene therapy product, Zolgensma is the first and the only approved therapy for treatment of spinal muscular dystrophy. In fact, Zolgensma has received approval in more than 45 regions across the globe, treating over 3,400 patients worldwide.

Recent Developments:

In January 2024, Novartis signed a licensing deal with Voyager Therapeutics, paying USD 100 million upfront to develop gene therapies.

In March 2023, Novartis Gene therapies secured exclusive, worldwide licenses with Nationwide Children's Hospital, REGENXBIO, and Généthon for the treatment of SMA using AAV9 gene therapy

Further details on recent initiatives taken by Novartis and other gene therapy companies have been provided in detail in the report.

Sibiono is Actively Involved in Clinical Trials Aimed at Further Validating the Efficacy of Gendicine and Exploring New Gene Therapy Techniques

8. Sibiono GeneTech

Sibiono GeneTech is a China-based biopharmaceutical company recognized for its remarkable contributions in the field of genetic medicine. The company is widely renowned for its milestone development for the world’s first commercially available gene therapy, Gendicine, an adenovirus-based recombinant human p53 therapy used for the treatment of oncological indications.

Sibiono GeneTech: Company Overview

Particulars	Specifications
Company Logo
Year of Establisment	1998
Number of Employees	1,001-5,000
Headquarters	China

The firm owns more than five invention patents related to the therapeutic application and production process of Gendicine. Additionally, the firm has consistently focused on expanding its R&D efforts in order to advance gene therapy technologies. Moreover, the firm is dedicated to leverage its scientific expertise to develop innovative treatments for a range of diseases, aiming to improve therapeutic outcomes for the patients.

Recent Developments:

In January 2023, Sibiono collaborated with academic institutions and research organizations in order to address challenges such as resistance to repeated administrations of rAd-p53 and to optimize therapeutic strategies for better patient outcomes.

Further details on recent initiatives taken by Sibiono GeneTech and other gene therapy companies have been provided in detail in the report.

What About Other Gene Therapy Companies Around the World?

The above presentation features eight top gene therapy developers selected from a pool of over 240 developers that we have compiled. If you're interested, you can download the Sample Report on this topic by Roots Analysis. For personalized assistance in identifying the most relevant solutions based on your specific criteria, please don't hesitate to reach out to us at sales@rootsanalysis.com.