Rankings: 28 Premier Ex Vivo Gene Therapy Establishments
Rankings: 28 Premier Ex Vivo Gene Therapy Establishments
The ex vivo gene therapy industry is a fast-growing segment within biotechnology that focuses on rectifying genetic disorders by modifying genes in cells outside the patients' bodies. Companies, such as bluebird bio and CRISPR Therapeutics, offer novel solutions to fight severe genetic diseases, revolutionizing the landscape of medical treatments. As science evolves, these companies continue to innovate, leveraging on technologies like CRISPR/Cas9 gene editing to develop transformative healthcare solutions. The industry is poised for a significant future expansion as it continues to tap upon the potential of gene manipulation.
Top 28 Ex Vivo Gene Therapy Companies
1. bluebird bio
- Website: bluebirdbio.com
- Headquarters: Cambridge, Massachusetts, United States
- Founded: 1993
- Headcount: 1001-5000
- Latest funding type: Post Ipo Equity
Bluebird is a biotechnology company founded in 2010, specializing in ex-vivo gene therapy. With a focus on severe genetic diseases, they have industry-leading programs for sickle cell disease, β-thalassemia, and cerebral adrenoleukodystrophy.
2. Intellia Therapeutics, Inc.
- Website: intelliatx.com
- Headquarters: Cambridge, Massachusetts, United States
- Founded: 2014
- Headcount: 201-500
- Latest funding type: Post Ipo Equity
Intellia Therapeutics is a biotechnology company focused on developing CRISPR/Cas9 gene editing therapies. They are dedicated to transforming the lives of patients suffering from genetic diseases. Intellia Therapeutics is committed to using their expertise in genome editing to create innovative treatments.
3. CRISPR Therapeutics
- Website: crisprtx.com
- Headquarters: Cambridge, Massachusetts, United States
- Founded: 2013
- Headcount: 201-500
- Latest funding type: Grant
CRISPR Therapeutics is a company that specializes in gene editing and cell therapies. They use the CRISPR/Cas9 therapeutic approach to treat genetically-defined diseases by editing cells inside the body.
4. Kite Pharma
- Website: kitepharma.com
- Headquarters: Santa Monica, California, United States
- Founded: 2009
- Headcount: 1001-5000
- Latest funding type: Acquired
Kite Pharma is a leading company in cancer immunotherapy, specializing in engineered T cell therapy. They aim to revolutionize cancer treatment by utilizing genetically modified immune cells to target tumors. Kite offers potentially lifesaving therapies for patients with hematologic cancers and solid tumors, including allogeneic therapies. With a focus on curative treatments, Kite is dedicated to bringing innovative solutions to those in need.
5. uniQure
- Website: uniqure.com
- Headquarters: Amsterdam, 1105Bp, Netherlands
- Founded: 1998
- Headcount: 201-500
- Latest funding type: Post Ipo Equity
uniQure is a global leader in gene therapy, specializing in delivering breakthrough treatments that transform patients' lives through the power of genomic medicine.
6. Orchard Therapeutics
- Website: orchard-tx.com
- Headquarters: London, England, United Kingdom
- Founded: 2015
- Headcount: 201-500
- Latest funding type: Post Ipo Equity
Orchard Therapeutics is a healthcare company specializing in genetic and severe disease treatments. They focus on discovering, developing, and commercializing new treatments using hematopoietic stem cell (HSC) gene therapy. By genetically modifying a patient's own blood stem cells, they aim to correct the underlying cause of disease in a single treatment.
7. REGENXBIO Inc.
- Website: regenxbio.com
- Headquarters: Rockville, Maryland, United States
- Founded: 2008
- Headcount: 201-500
- Latest funding type: Post Ipo Equity
REGENXBIO is a company dedicated to discovering and developing innovative gene therapies to address severe and debilitating diseases with unmet needs. They aim to improve treatment options and provide curative potential through their gene therapy solutions. With their NAV® Technology Platform, they are driving progress towards making gene therapy a reality for patients.
8. Poseida Therapeutics, Inc.
- Website: poseida.com
- Headquarters: San Diego, California, United States
- Founded: 2014
- Headcount: 201-500
- Latest funding type: Post Ipo Equity
Poseida Therapeutics is a clinical-stage biopharmaceutical company specializing in cell and gene therapies for the treatment of cancer and rare diseases.
9. EdiGene Inc.
- Website: edigene.com
- Headquarters: Beijing, Beijing, China
- Founded: 2015
- Headcount: 201-500
- Latest funding type: Series B
EdiGene is a company that specializes in genome editing technologies for the treatment of genetic diseases and cancer. They translate these innovative technologies into transformative therapies, offering new hope and options for patients in China and worldwide.
10. Prime Medicine, Inc.
- Website: primemedicine.com
- Headquarters: Cambridge, Massachusetts, United States
- Founded: 2019
- Headcount: 51-200
- Latest funding type: Ipo
Prime Medicine is a biotechnology company that specializes in prime editing, a next-generation gene editing approach. Their technology aims to address the genetic cause of disease and provide patients with long-lasting cures. By precisely editing faulty DNA sequences in the genome, Prime Medicine offers a potential solution for a wide range of therapeutic applications.
11. Cellectis
- Website: cellectis.com
- Headquarters: Paris, Ile-De-France, France
- Founded: 1999
- Headcount: 51-200
- Latest funding type: Post Ipo Equity
Cellectis is a clinical-stage biopharmaceutical company focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells. They offer off-the-shelf immunotherapies that can work for a large number of patients.
12. SalioGen Therapeutics
- Website: saliogen.com
- Headquarters: Cambridge, Massachusetts, United States
- Founded: 2020
- Headcount: 51-200
- Latest funding type: Series B
SalioGen is a genetic medicine company focused on developing therapies for inherited disorders. They utilize their innovative Gene Coding approach, which involves adding new genetic codes to modify the function of genes. With their Exact DNA Integration Technology (EDIT) platform, they have the potential to revolutionize the treatment paradigm for inherited disorders.
13. Arbor Biotechnologies
- Website: arbor.bio
- Headquarters: Cambridge, Massachusetts, United States
- Founded: 2016
- Headcount: 11-50
- Latest funding type: Series B
Arbor Biotechnologies is a biotechnology company focused on pioneering gene editing technology to develop functional cures for genetic diseases, with a specific emphasis on liver and CNS diseases.
14. Life Edit Therapeutics
- Website: lifeeditinc.com
- Headquarters: Research Triangle Park, North Carolina, United States
- Founded: 2017
- Headcount: 11-50
- Latest funding type: Acquired
Life Edit Inc. is a company that specializes in gene editing therapies for patients with genetic diseases. They are dedicated to making innovative and curative treatments accessible to those in need. With a focus on discovery and unlimited potential, they aim to rewrite the future of gene editing by providing versatile options for targeting genetic disorders.
15. Scribe Therapeutics
- Website: scribetx.com
- Headquarters: Alameda, California, United States
- Founded: 2017
- Headcount: 11-50
- Latest funding type: Series B
Scribe is a molecular engineering company specializing in genetic medicine. They have developed highly active editing activities capable of editing out the underlying causes of disease. Join their mission to engineer the next 20 years of gene editing breakthroughs.
16. Avrobio
- Website: avrobio.com
- Headquarters: Cambridge, Massachusetts, United States
- Founded: 2015
- Headcount: 51-200
- Latest funding type: Ipo
AVROBIO is a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease. They are focused on targeting lysosomal disorders and developing personalized therapies for patients. Their goal is to enable individuals with genetic diseases to live longer and free from disease progression.
17. Flash Therapeutics
- Website: flashtherapeutics.com
- Headquarters: Toulouse, Occitanie, France
- Founded: 2018
- Headcount: 11-50
- Latest funding type: Debt Financing
Flash Therapeutics is a company that specializes in providing purified and highly concentrated particles for gene therapy. With their expertise in lentiviral vector manufacturing, they support projects from research to GMP grade production. They offer R&D and GMP manufacturing services and have partnered programs in regenerative medicine and cell therapy.
18. Tessera Therapeutics
- Website: tesseratherapeutics.com
- Headquarters: Cambridge, Massachusetts, United States
- Founded: 2018
- Headcount: 51-200
- Latest funding type: Series C
Tessera Therapeutics is a pioneering company specializing in Gene Writing™, a new genome engineering technology that permanently alters the human genome to treat diseases at their source.
19. Avectas
- Website: avectas.com
- Headquarters: Kildare, Ireland, United States
- Founded: 2011
- Headcount: 11-50
- Latest funding type: Grant
Avectas is a cell engineering technology business focused on improving the cost, manufacturing, and patient outcomes for the next generation of cellular therapies.
20. OZ Biosciences
- Website: ozbiosciences.com
- Headquarters: Marseille, Paca, France
- Founded: 2003
- Headcount: 11-50
OZ Biosciences is a biotech company specializing in the development and production of innovative tools and reagents for transfection and gene therapy. They offer a wide range of products including transfection reagents, protein delivery systems, vaccine adjuvants, and genome editing tools. With a focus on quality and efficiency, OZ Biosciences aims to support researchers and scientists in their quest for new discoveries and advancements in biotechnology.
21. Hopewell Therapeutics Inc.
- Website: hopewell-tx.com
- Headquarters: Woburn, Massachusetts, United States
- Founded: 2018
- Headcount: 11-50
- Latest funding type: Series Unknown
Hopewell is a biotechnology company that specializes in the development of lipid nanoparticles (LNPs) for efficient and targeted delivery of nucleic acid-based cargos. Their mission is to solve the challenges associated with drug discovery by providing safe and selective delivery systems. They offer cutting-edge technology and services in the fields of immunotherapy, gene therapy, and gene editing.
22. Genespire
- Website: genespire.com
- Headquarters: Milan, Lombardy, Italy
- Founded: 2020
- Headcount: 1-10
- Latest funding type: Series A
Genespire is a biotechnology company focused on the development of durable and transformative gene therapies for patients affected by genetic diseases, particularly primary immunodeficiencies and inherited metabolic diseases. Based in Milan, Italy, Genespire was founded in March 2020 by the gene therapy pioneer Prof. Luigi Naldini, Dr. Alessio Cantore, Fondazione Telethon and Ospedale San Raffaele. It is a spin-off of SR-Tiget, a world leading cell and gene therapy research institute and is backed by Sofinnova Partners.
23. Strand Therapeutics
- Website: strandtx.com
- Headquarters: Cambridge, Massachusetts, United States
- Founded: 2017
- Headcount: 1-10
- Latest funding type: Series A
Strand Therapeutics is an emerging biopharmaceutical company applying synthetic biology to RNA therapeutics. They are developing a platform for the creation of programmable, long-acting mRNA drugs capable of delivering precise, multi-functional, potentially curative treatments with a single dose.
24. Ensoma
- Website: ensoma.com
- Headquarters: Boston, Massachusetts, United States
- Founded: 2019
- Headcount: 51-200
- Latest funding type: Series B
Ensoma is a company at the forefront of medical innovation, focusing on developing one-time in vivo treatments to cure diseases by engineering cells of the hematopoietic system. They have a strategic collaboration with Takeda and are supported by top-tier investors. Their pioneering Engenious™ platform enables them to tackle a wide range of diseases, including cancer, autoimmune diseases, and inherited conditions.
25. ElevateBio
- Website: elevate.bio
- Headquarters: Waltham, Massachusetts, United States
- Founded: 2017
- Headcount: 201-500
- Latest funding type: Angel
Elevate.bio is a company that integrates cutting-edge technologies, end-to-end capabilities, and the smartest people to regenerate cells, rewrite genes, and redefine manufacturing. They provide next-generation enabling technologies in gene editing, induced pluripotent stem cells (iPSCs), and cell, protein, and vector engineering to equip partners with tools for potentially groundbreaking cell and gene therapies.
26. Ixaka
- Website: ixaka.com
- Headquarters: London, England, United Kingdom
- Founded: 2015
- Headcount: 51-200
- Latest funding type: Series Unknown
Ixaka is a cell and gene therapy company focused on utilizing the natural power of the body to cure a wide range of serious diseases. Their proprietary technologies, concentrated Multi-Cell Therapies (MCT) and Nano-Particle Therapeutics (NPT), have the potential to treat and even cure cancers, cardiovascular diseases, genetic disorders, neurological and ocular diseases.
27. Beam Therapeutics
- Website: beamtx.com
- Headquarters: Cambridge, Massachusetts, United States
- Founded: 2017
- Headcount: 501-1000
- Latest funding type: Post Ipo Equity
Beam Therapeutics is a cutting-edge biotech company focused on base editing applications, delivering clinically validated technologies for gene-based editing. They offer a suite of innovative, nonviral and viral delivery modalities to advance the field of genetic medicine.
28. Sangamo Therapeutics, Inc.
- Website: sangamo.com
- Headquarters: Brisbane, California, United States
- Founded: 1995
- Headcount: 201-500
- Latest funding type: Post Ipo Equity
Sangamo is a biotechnology company that specializes in using zinc finger technology to develop genomic cures for severe diseases. Their expertise allows them to create treatments for patients who currently only have access to symptom management. They have a strong commitment to patient care and offer resources and support for both patients and healthcare professionals.
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