Top 23 Companies in Monogenic Disease Gene Therapy
Top 23 Companies in Monogenic Disease Gene Therapy
The industry landscape consists of diverse companies that are at the forefront of genetic therapies for monogenic diseases. These companies focus on the production and development of novel treatments that are transforming the healthcare sector, particularly for rare genetic conditions with high unmet medical needs. Emphasizing the genetic modification via gene therapy, these organizations seek to understand and decipher the complex underpinning of monogenic disorders. The industry is moving progressively towards expanding treatment options, optimizing gene delivery platforms, and enhancing the benefit-risk profiles for patients.
Top 23 Monogenic Disease 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. Ultragenyx
- Website: ultragenyx.com
- Headquarters: Novato, California, United States
- Founded: 2010
- Headcount: 501-1000
- Latest funding type: Post Ipo Equity
Ultragenyx is a biopharmaceutical company focused on developing and commercializing novel treatments for rare genetic diseases. They have a diverse portfolio of approved therapies and product candidates targeting diseases with high unmet medical need. Their goal is to provide safe and effective therapies to patients with a sense of urgency.
3. 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.
4. 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.
5. Taysha Gene Therapies
- Website: tayshagtx.com
- Headquarters: Dallas, Texas, United States
- Founded: 2020
- Headcount: 51-200
- Latest funding type: Post Ipo Equity
Taysha Gene Therapies is a company focused on developing gene therapies for monogenic CNS diseases. They aim to eradicate these diseases by discovering, developing, and commercializing novel gene therapies. With a pipeline of gene therapies, they are committed to being an ally to the rare disease community.
6. I-Stem
- Website: istem.eu
- Headquarters: Evry Cedex, Ile-De-France, France
- Founded: 2005
- Headcount: 51-200
I-Stem is an institute specializing in stem cell research for the treatment and study of monogenic diseases.
7. Passage Bio
- Website: passagebio.com
- Headquarters: Philadelphia, Pennsylvania, United States
- Founded: 2017
- Headcount: 51-200
- Latest funding type: Post Ipo Equity
Passage Bio is a biotechnology company specializing in AAV gene therapies for CNS diseases. They focus on developing treatments for GM1 Gangliosidosis, Krabbe Disease, and Frontotemporal Dementia. Their commitment lies in providing patient and community resources to improve the lives of patients and their families. They also engage in collaboration and share their scientific advancements through partnerships with Penn GTP and innovative manufacturing practices.
8. Atsena Therapeutics
- Website: atsenatx.com
- Headquarters: Durham, North Carolina, United States
- Founded: 2019
- Headcount: 11-50
- Latest funding type: Series A
Atsena Therapeutics is a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness. They have ongoing clinical trials evaluating gene therapies for various monogenic eye diseases, including Leber congenital amaurosis (LCA1) and X-linked retinoschisis (XLRS). Their innovative adeno-associated virus (AAV) technology platform allows them to overcome the challenges presented by inherited retinal diseases.
9. LEXEO Therapeutics
- Website: lexeotx.com
- Headquarters: New York, New York, United States
- Founded: 2018
- Headcount: 11-50
- Latest funding type: Series B
LEXEO is a gene therapy company that develops investigational therapies for rare monogenic diseases and diseases affecting prevalent patient populations. They utilize engineered adeno-associated viruses to transfer therapeutic genes to patients, targeting genetically defined cardiovascular and central nervous system diseases. LEXEO's therapies have potential in treating Friedreich's Ataxia, Alzheimer's, and other similar conditions.
10. Cato Research
- Website: cato-sms.com
- Headquarters: Durham, North Carolina, United States
- Founded: 1988
- Headcount: 201-500
CATO SMS is a global company offering regulatory and development consulting services in the pharmaceutical industry. With a focus on strategic consulting, pharmacokinetics/pharmacodynamics (PK/PD), pharmacometrics, and biosimulation, CATO SMS helps clients navigate the complexities of drug development. Their team of experienced consultants provides expertise in various therapeutic areas, including oncology & hematology, rare diseases & orphan drugs, and cell and gene therapy.
11. Discovery Labs
- Website: thediscoverylabs.com
- Headquarters: King Of Prussia, Pennsylvania, United States
- Founded: 2019
- Headcount: 51-200
The Discovery Labs offers laboratory and cGMP manufacturing space for the discovery and development of life-saving therapies and medicines.
12. Purespring Therapeutics
- Website: purespringtx.com
- Headquarters: London, England, United Kingdom
- Founded: 2020
- Headcount: 11-50
- Latest funding type: Series A
Purespring Therapeutics is leading a revolution in the treatment of kidney diseases by providing transformative therapies. They aim to bring disease-modifying treatments to patients suffering from chronic kidney diseases, including rare monogenic diseases and common forms of kidney disease. Their mission is to stop kidney diseases in their tracks and improve the quality of life for patients.
13. BioViva USA Inc
- Website: bioviva-science.com
- Headquarters: Bainbridge Island, Washington, United States
- Founded: 2014
- Headcount: 11-50
- Latest funding type: Equity Crowdfunding
BioViva is a gene therapy company that focuses on lengthening healthy human lifespans. They develop innovative platforms to treat severe genetic disorders and cellular aging.
14. Clinical OMICs
- Website: clinicalomics.com
- Headquarters: Ny, New Rochelle, United States
- Founded: 2014
- Headcount: 51-200
Clinical Omics offers products and solutions in precision medicine, translational research, molecular diagnostics, and oncology/therapeutics. They provide various genetic and biotechnology based services and cater to healthcare industry.
15. Brooklyn ImmunoTherapeutics
- Website: brooklynitx.com
- Headquarters: Brooklyn, New York, United States
- Founded: 1994
- Headcount: 11-50
- Latest funding type: Ipo
Brooklyn ImmunoTherapeutics is a company focused on immuno-oncology and developing innovative therapies for the treatment of cancer. They specialize in the discovery, development, and commercialization of novel immunotherapies.
16. Jasper Therapeutics
- Website: jaspertherapeutics.com
- Headquarters: Redwood City, California, United States
- Founded: 2018
- Headcount: 11-50
- Latest funding type: Post Ipo Equity
Jasper Therapeutics is a biotechnology company specializing in stem cell grafts and mRNA therapies. They focus on developing innovative treatments for various diseases and conditions. Through their cutting-edge research and scientific expertise, Jasper Therapeutics aims to revolutionize the field of regenerative medicine.
17. Schwitz Biotech
- Website: schwitzbiotech.com
- Headquarters: Ahmedabad, Gujarat, India
- Founded: 1998
- Headcount: 51-200
Schwitzbiotech is a company that specializes in regenerative medicine and stem cell therapies for repairing damaged organs.
18. Genemedi Biotech
- Website: genemedi.net
- Headquarters: Shanghai, Shanghai, China
- Founded: 2012
- Headcount: 11-50
GeneMedi offers a wide range of products and services for biologics discovery, such as CRISPR/Cas9 AAV production, pre-made lentivirus particles, pre-made antibody & protein & conjugate products, and veterinary diagnostics by species.
19. Lemonex
- Website: lemonexbio.com
- Headquarters: Seoul, Seoul, Korea (the Republic of)
- Founded: 2013
- Headcount: 11-50
Lemonexbio is a global biocerapeutics and biotechnology company that develops innovative next-generation drug delivery and biosensor technology for use in immunotherapy, monoclonal antibodies, gene therapy, and synthetic drugs.
20. Cell & Gene Therapy Showcase
- Website: cellgenetherapy.com
- Headquarters: London, England, United Kingdom
- Founded: 2007
- Headcount: 51-200
CellGene Therapy is a biotechnology company that develops innovative cell and gene therapies to treat various diseases. Their therapies focus on repairing or replacing damaged cells and genes to restore normal cellular function.
21. Cerba Research
- Website: cerbaresearch.com
- Headquarters: Ghent, Flemish Region, Belgium
- Founded: 1984
- Headcount: 1001-5000
- Latest funding type: Acquired
Cerba Research is a global research organization specializing in medical device and diagnostics solutions. With a focus on next-generation sequencing for hematological malignancies, they offer a range of services including clinical trial operations, flow cytometry, genomics, and specialty lab solutions.
22. Novellus
- Website: novellustx.com
- Headquarters: Cambridge, Massachusetts, United States
- Founded: 2014
- Headcount: 11-50
- Latest funding type: Acquired
Novellus Therapeutics is a biotechnology company that develops innovative cell therapies to treat various diseases using cutting-edge cell engineering technologies.
23. Eterna Therapeutics
- Website: eternatx.com
- Headquarters: San Diego, California, United States
- Founded: 1994
- Headcount: 11-50
Eterna Therapeutics is a company that focuses on cell engineering for personalized, precision medicine.
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