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Novartis Gene Therapies, until 2020 known as AveXis, is a biotechnology company that develops treatments for rare neurological genetic disorders. It was founded in Dallas , Texas , United States in 2012 by John Carbona after reorganizing a company called BioLife Cell Bank founded by David Genecov and John Harkey.
Fidanacogene elaparvovec, previously known by its study ID number SPK-9001, [12] is a gene therapy for the treatment of hemophilia B. It was developed by Spark in partnership with Pfizer . Fidanacogene elaparvovec is an adeno-associated viral vector which is designed to transfer a working copy of the Factor IX gene into the livers of patients ...
On Monday, Sangamo Therapeutics, Inc. (NASDAQ:SGMO) regained the development and commercialization rights to giroctocogene fitelparvovec, an investigational gene therapy product candidate for ...
BioViva was founded in 2015. [2] The CEO, Elizabeth Parrish, started the company by becoming the first person in human history to take gene therapies associated with lengthening lifespans in model organism, [3] she appeared at WIRED Health 2017 in London to discuss BioViva's testing of gene therapies targeting hallmarks of the ageing process.
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PTC Therapeutics’ therapy, known as PTC518, is an oral medication designed to reduce the production of the mutant protein linked to the genetic mutation responsible for the disease. [15] This mutant protein is believed to contribute to neuronal death, driving disease progression. [ 16 ]
Sarepta Therapeutics, Inc. is a medical research and drug development company with corporate offices and research facilities in Cambridge, Massachusetts, United States.. Incorporated in 1980 as AntiVirals, [2] shortly before going public the company changed its name from AntiVirals to AVI BioPharma soon with stock symbol AVII and in July 2012 changed name from AVI BioPharma to Sarepta ...
Kite Pharma, Inc. is an American biotechnology company that develops cancer immunotherapy products with a primary focus on genetically engineered autologous CAR T cell therapy - a cell-based therapy which relies on chimeric antigen receptors and T cells.