Prominent 23 In Vivo Gene Therapy Enterprises
Prominent 23 In Vivo Gene Therapy Enterprises
The industry under discussion houses companies working in the field of in vivo gene therapy, a burgeoning medical domain that aims to treat or prevent diseases by inserting, altering or removing genes within the patient's body. This radical technique offers the potential for transformative treatments of numerous genetic diseases, from rare genetic disorders to more widespread issues like cancer. The firms in this industry, such as BioMarin Pharmaceutical Inc., use advanced technologies like CRISPR for genetic innovation, aiming to provide patients with significantly improved lives. As society continues to demand better healthcare solutions, the industry is expected to grow further.
Top 23 In Vivo Gene Therapy Companies
1. BioMarin Pharmaceutical Inc.
- Website: biomarin.com
- Headquarters: San Rafael, California, United States
- Founded: 1997
- Headcount: 1001-5000
- Latest funding type: Post Ipo Debt
BioMarin is a pharmaceutical company specializing in genetic therapies. They develop and provide transformative treatments for various genetic diseases, such as Morquio A Syndrome, PKU, and MPS. With a focus on gene therapy research, BioMarin aims to maximize the impact of genetic innovation and improve patients' lives.
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. 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.
5. Editas Medicine
- Website: editasmedicine.com
- Headquarters: Cambridge, Massachusetts, United States
- Founded: 2013
- Headcount: 51-200
- Latest funding type: Post Ipo Equity
Editas Medicine is a biotechnology company specializing in CRISPR gene editing. They offer innovative solutions for patients and focus on developing cutting-edge therapies. With a strong research pipeline and a dedicated team, Editas Medicine aims to revolutionize the field of genetic 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. Precision BioSciences, Inc.
- Website: precisionbiosciences.com
- Headquarters: Durham, North Carolina, United States
- Founded: 2006
- Headcount: 201-500
- Latest funding type: Post Ipo Equity
Precision BioSciences is a company dedicated to in vivo gene editing therapies. They specialize in permanently correcting genetic diseases through their ARCUS platform and CAR T immunotherapy. Precision BioSciences collaborates with healthcare companies and researchers to develop potentially curative treatments. They offer expertise in precision medicine and have a strong skill set within the genome editing industry.
8. 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.
9. 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.
10. Homology Medicines, Inc.
- Website: homologymedicines.com
- Headquarters: Bedford, Massachusetts, United States
- Founded: 2015
- Headcount: 51-200
- Latest funding type: Post Ipo Equity
Homology Medicines is a genetic medicine company dedicated to developing new treatments and cures for rare diseases. With their powerful in vivo genetic medicines platform, they aim to transform the lives of patients by potentially offering them a chance at a cure.
11. 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.
12. 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.
13. 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.
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. 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.
17. Carmine Therapeutics
- Website: carminetherapeutics.com
- Headquarters: Cambridge, Massachusetts, United States
- Founded: 2019
- Headcount: 11-50
- Latest funding type: Series A
Carmine Therapeutics is a company that is pioneering powerful new genetic medicines based on red blood cell extracellular vesicles (RBCEVs). They are developing next-generation non-viral gene therapies that overcome the limitations of viral-based gene therapies. Their proprietary Red Cell EV Gene Therapy (REGENT®) platform generates a pipeline of next-generation disease-modifying gene therapies for a broad spectrum of diseases.
18. Enochian BioSciences Inc
- Website: enochianbio.com
- Headquarters: Los Angeles, California, United States
- Founded: 2011
- Headcount: 11-50
- Latest funding type: Post Ipo Equity
Enochian BioSciences is a biopharmaceutical company specializing in the identification, development, manufacturing, and commercialization of gene-modified cell therapy. They offer a gene-modified cell therapy platform that can be used for various indications, including HIV/AIDS and Oncology.
19. 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.
20. StrideBio, Inc
- Website: stridebio.com
- Headquarters: Durham, North Carolina, United States
- Founded: 2015
- Headcount: 51-200
- Latest funding type: Series B
StrideBio is a gene therapy company that develops best-in-class treatments for life-threatening diseases. They specialize in novel capsids with improved functionalities and potential for better safety.
21. Epigeneres Biotech
- Website: epigeneres.com
- Headquarters: Mumbai, Maharashtra, India
- Founded: 2008
- Headcount: 11-50
- Latest funding type: Series B
Epigeneres is a leading biotechnology company specializing in advanced genetic therapeutics, tissue regeneration, and reversal of arteriosclerosis. They discover, develop, manufacture, and commercialize medicines for the treatment and prevention of serious medical conditions and age-related diseases.
22. Holostem Terapie Avanzate S.r.l.
- Website: holostem.com
- Headquarters: Modena, Emilia-Romagna, Italy
- Founded: 2008
- Headcount: 11-50
Holostem Terapie Avanzate is a biotechnology company specializing in the development, production, registration, and distribution of advanced therapy medicinal products (ATMPs) based on epithelial stem cell cultures for cellular and genetic therapy.
23. Applied StemCell
- Website: appliedstemcell.com
- Headquarters: Milpitas, California, United States
- Founded: 2008
- Headcount: 51-200
- Latest funding type: Series D
Applied StemCell is a biotechnology company that specializes in providing various stem cell products and services. They offer off-the-shelf mouse models, iPSC and mESC lines, genome edited iPSCs, differentiated cells, MEF feeder cells, and iPSC generation kits. Additionally, they provide CRO/CDMO services, cell therapy manufacturing, in vivo assay services, and mouse model generation services. Located in Milpitas, California, they aim to support research and development in the field of regenerative medicine and drug discovery.
Want to find more in vivo gene therapy companies?
If you want to find more companies that provide transformative treatments for genetic diseases you can do so with Inven. This list was built with Inven and there are hundreds of companies like these globally.
With Inven you'll also get to know the company's:
- Ownership: Which of these are private equity backed? Which are family-owned?
- Contact data: Who are the owners and CEO's? What are their emails and phone numbers?
- Financials: How do these companies perform financially? What are their revenues and profit margins?
...and a lot more!
