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Gene Therapy Companies

Gene therapy is an innovative field that involves the introduction, modification, or correction of genetic material to treat or prevent diseases. Gene therapy companies focus on developing and commercializing gene-based treatments for various medical conditions.

Gene Therapy Key CompaniesLatest Gene Therapy Companies Update

November 2023: The Phase 1 REGENERATE MSA-101 clinical trial of AB-1005, a gene therapy aimed at treating multiple system atrophy-parkinsonian type (MSA-P), has recently commenced at the Ohio State University Wexner Medical Center. Asklepios BioPharmaceutical, Inc., a gene therapy company that operates as a subsidiary of Bayer AG, has announced the randomization of the first patient in this trial. This achievement represents a noteworthy advancement in the progress of AB-1005 gene therapy, which utilizes an adeno-associated viral vector known as AAV2-GDNF to encode glial cell line-derived neurotrophic factor. The delivery of this vector to the putamen region puts this therapeutic approach closer to its possible application in treating patients. The investigation of AB-1005 for the treatment of mild to moderate Parkinson's disease is now underway, with the completion of recruitment in the Phase 1b research.


November 2023: the Medicines and Healthcare Products Regulatory Agency of the United Kingdom issued conditional marketing authorization to Casgevy, a gene editing medication developed by Vertex Pharmaceuticals and CRISPR Therapeutics. This milestone represents the first approval of a therapy utilizing CRISPR-based gene editing technology. The authorization of Casgevy for the treatment of sickle cell anemia (SCA) and β-thalassaemia marks a significant milestone in the field of genetic diseases, signifying the emergence of a promising age in which the possibility of curing inherited problems becomes a viable prospect. This development brings hope to millions of individuals worldwide who are currently facing the challenges posed by these hereditary ailments. The primary constraint impeding the extensive use of this medication is its exorbitant expense. According to estimations, the projected cost per patient is roughly $2 million.List of Gene Therapy Key companies in the market

  • Amgen Inc. (US)

  • Spark Therapeutics Inc. (US)

  • Bristol-Myers Squibb Company (US)

  • Novartis AG (Switzerland)

  • Gilead Sciences Inc. (US)

  • Celgene Corporation (US)

  • Thermo Fisher Scientific Inc. (US)

  • CEVEC (Germany)

  • uniQure N.V. (Netherlands)

  • Orchard Therapeutics plc (UK)

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