Top 10 Gene Therapy Companies in the World
Are You Aware of the fact that over 350 million people worldwide live with rare genetic disorders, as per data from the National Human Genome Research Institute?
Most of us might see it as just another statistical data, but behind every single number lies a human life that is getting impacted due to some abnormalities in their gene, which has no cure. A minor change in a single gene can turn someone’s life upside down, leading to disorders like fibrosis, Huntington’s disease, and sickle cell anemia, to name a few. The list is enormous. These genetic changes are often hereditary, passed down from one generation to another.
Now, imagine a world where these genetic errors can be corrected! A world where millions of lives can be saved by fixing the abnormalities in their DNA? Thanks to global advancements in science, this dream is becoming a reality through gene therapy.
Thanks to the advancement of technology, Gene therapy has emerged as a lifesaver. It doesn’t just treat symptoms. It cures the disease from its root cause. They can potentially have long-lasting effects and, in some cases, permanent cures too. All thanks to these
companies who are on their way to rewrite the very code of life, transforming millions of lives, and ending the generational curse.Let’s look at the TOP 10 Companies That are working in the field of Gene Therapy Research.
- Transforming lives with Bluebird Bio
With a mission of transforming the lives of people suffering from genetic disorders, Bluebird Bio was founded in 2010. They are pursuing curative gene therapies for sickle cell disease, β-thalassemia, and cerebral adrenoleukodystrophy. With its largest and deepest ex-vivo gene therapy data set worldwide, Bluebird Bio has become a global leader in gene therapy. They have three distinct investigational gene therapies that utilize gene addition combined with an autologous hematopoietic stem cell transplant (HSCT).
Their one-time gene therapy, Zynteglo, is for treating adult and pediatric patients with beta-thalassemia. This personalized therapy uses the patient’s own blood stem cells and adds functional copies of the beta-globin gene to the cells. Another FDA-approved Skysona, the company’s most promising gene therapy for treating boys of age group 4 to 17 who are suffering from cerebral adrenoleukodystrophy (CALD). They are currently developing and delivering new therapies for severe genetic diseases that have limited or no treatment options.
2. Innovation at its best: Sangamo Therapeutics
Founded in 1995, Sangamo has made a significant impact in the development of gene therapy and genomic medicine. The work on developing groundbreaking discoveries that act on the root cause of the genetic disorders. Their cutting-edge gene editing technology, Zinc Finger Nucleases (ZFN), has enabled scientists to correct the mutations responsible for these genetic disorders. With this technology, they are working on curing various disorders, including hemophilia and Fabry disease. In addition to this, they are also working on developing therapies for sickle cell disease and Huntington’s disorder, where they want to offer a potential long-term cure rather than just symptom management. These innovations not only give hope for a better future but also permanent treatment for these genetic disorders.
Top 10 Gene Therapy Companies
3. Restoring the Vision: Spark Therapeutics
Spark Therapeutics has established itself as a leader with their groundbreaking innovations. The company was founded in 2013, and later, it became a Member of the Roche Group in 2019. These two biotech giants have collaborated and worked on developing Luxturna. This gene therapy was developed to treat inherited blindness. Their integrated technology has transformed genes into medicine to treat genetic disorders, including inherited retinal diseases (IRDs), liver-directed diseases, hemophilia, and neurodegenerative diseases.
With the advancement in research, they have designed research protocols using genetically engineered adeno-associated viral (AAV) vectors. These encapsulate genetic material for the potential treatment. These vectors are selected through several preclinical testing and clinical trial validations. AAV has minimal pathogenicity and the ability to establish long-term gene expression in different tissues. This makes it an emerging pivotal delivery tool in clinical gene therapy ( Wang, JH. et al., 2024).
4. Future of Precision Genomic Medicines: Editas Medicine
Editas Medicine is a clinical-stage gene editing company determined to translate the power and potential of CRISPR/Cas12a and CRISPR/Cas9 genome editing systems into treatments that will transform gene therapy. They aim to discover, develop, and commercialize precision genomic medicines for various diseases. They hold exclusive licensees of Broad Institute‘s Cas12a patent estate and Broad Institute and Harvard University’s Cas9 patent estates for human medicines. The company mainly focuses on rare eye diseases such as Leber congenital amaurosis (LCA10). This eye disease causes vision loss or blindness from birth or during early childhood.
The FDA has granted Rare Pediatric Disease and Orphan Drug designations for EDIT-101. The company developed a CRISPR/Cas9-based experimental medicine to treat Leber congenital amaurosis 10 (LCA10) by deleting the IVS26 CEP290 mutant allele. It was also designed as an Orphan Medicinal Product designation from the European Medicines Agency (EMA).
5. Unlocking the Future of Gene Therapy: CRISPR Therapeutics
The revolutionary gene-editing tool by Dr. Emmanuelle Charpentier and Dr. Jennifer Doudna was the first step in establishing CRISPR Therapeutics. Dr. Charpentier, along with Rodger Novak and Shaun Foy, founded the company in 2013. The company is working on translating the CRISPR/Cas9 discovery into potential therapeutics. With their collaboration with Vertex Pharmaceuticals Incorporated, they now work as a co-developer of CASGEVY™ (Hoy S. M., 2024). This is the first-ever CRISPR-based gene-editing treatment. Currently, this treatment is approved in countries such as the United States, Great Britain, and Bahrain for certain eligible patients suffering from sickle cell disease or transfusion-dependent beta-thalassemia.
6. Investigator of Gene Therapy: UniQure
As stated in 1998, with the name Amsterdam Molecular Therapeutics, UniQure was the first to get approval for its gene therapy.UniQure is working on delivering a single treatment with potentially curative results. They are advancing a pipeline of proprietary gene therapies for treating Huntington’s disease, refractory temporal lobe epilepsy, Fabry disease, ALS, and other diseases. The company has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to AMT -191, an investigational gene therapy for treating Fabry disease.
AMT-191 is a one-time intravenously administered investigational AAV5-based gene therapy that uses a proprietary, highly potent promoter to deliver a GLA transgene designed to target the liver to produce GLA protein. Another gene therapy, AMT-061, also known as Etranacogene dezaparvovec, is a promising gene therapy to treat hemophilia B. The therapy was approved by the FDA in 2022. The clinical trials have shown promising results during the clinical trials.
7. Rewriting the Genetic Treatment: Pfizer
The multinational pharmaceutical company Pfizer is also among the Top 10 Gene Therapy Companies. It joined the revolution of rewriting the future of genetic treatment. They strive to set the standard for quality, safety, and value with their discoveries, development, and manufacture of healthcare products, such as innovative medicines and vaccines.
Their recent gene therapy, DURVEQTIX (also known fidanacogene elaparvovec), is a potential treatment for severe and moderately severe hemophilia B in adults without a history of factor IX inhibitors and without detectable antibodies to variant AAV serotype Rh 74. DURVEQTIX was approved by the US Food and Drug Administration (FDA) for hemophilia B as BEQVEZTM in April 2024. The therapy contains a bio-engineered adeno-associated virus (AAV) capsid and a high-activity variant of the human coagulation FIX gene. This was designed to enable hemophilia B patients to produce factor IX via a one-time dose. In December 2014, Pfizer licensed DURVEQTIX from Spark® Therapeutics.
8. Powerhouse in Biotech: Novartis
Since 2013, Novartis Gene Therapies, formerly AveXis, has been working on bringing change to those devastated by genetic diseases. Novartis gene therapy R&D currently focuses on AAV-based therapies and CRISPR-based technologies. They consider AAV as a safe and effective vector for gene therapies. On the other hand, they are conducting early research on CRISPR-based technologies across hematology and ophthalmology to potentially treat diseases by correcting genetic defects.
They have collaborated with Sangamo Therapeutics to develop gene therapies for neurodevelopmental diseases. This collaborated team will be working on identifying the potential therapies for several diseases, including autism spectrum disorder. Novartis has expanded its capabilities in several distinct gene therapy platforms. Some of these platforms are AAVs, Chimeric Antigen Receptor T-cells (CAR-Ts), and CRISPR. The company successfully launched Zolgensma in 2019 after the FDA approval. This gene therapy medicine is for children aged two and below who are suffering from spinal muscular atrophy (SMA).
9. Bringing Hope with Innovation: BioMarin Pharmaceutical
With the relentless pursuit to translate genetic discoveries into medicines, BioMarin Pharmaceuticals is one of its kind. This global biotech company is dedicated to transforming lives through its genetic discoveries. The company develops and manufactures targeted therapies that address the root cause of the genetic disorder. Their unparalleled research and development capabilities have resulted in eight groundbreaking commercial therapies that are helping millions of people fight against genetic disorders. Its distinctive approach to drug discovery has helped them understand the biology and provided an opportunity to be first-to-market over other existing treatments.
They have a diverse pipeline of commercial, pre-clinical, and clinical products that address significant unmet medical needs. They have announced that their product, VOXZOGO, is the first and only approved treatment for children with achondroplasia in the U.S., Japan, and Australia.
Top 10 Gene Therapy Companies
10. Unlocking the new Possibilities: Regenxbio
Kenneth T. Mills started Regenxbio in 2009 with a commitment to develop gene therapies that will improve the treatment options for people with unmet needs. In their early days, they focused on licensing their NAV® Technology Platform to other biotech companies. They were able to support other pharma and budding companies with their NAV® vectors. This platform currently consists of more than 100 novel AAV vectors, including AAV7, AAV8, and AAV9. The first gene therapy based on NAV® technology was approved by the US FDA for treating Spinal Muscular Atrophy in 2019.
These pioneering companies are working on transforming imagination into reality with their innovations and discoveries. A future where these diseases are not just managed but also cured at the genetic level. The contributions of these companies have given hope that the future of medicine is bright and gene therapy is no longer a textbook concept but a real-life solution. With gene therapy, we can cure genetic disorders, saving millions of lives.
Every clinical trial, every advanced therapy, and every breakthrough is taking us one step closer to breaking the generational cycle of inheritance forever. The biotech industry is at the forefront of innovation, which is reshaping the future of medicine. Let’s be part of the transformation and make a meaningful impact.
