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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 ...
Yahoo Finance's Julie Hyman discusses the reversal in Bluebird Bio shares after the FDA approved its second gene therapy.
bluebird bio, Inc., based in Somerville, Massachusetts, is a biotechnology company that develops gene therapies for severe genetic disorders. [1]The company's only - in the European Union (EU) - approved drug is betibeglogene autotemcel (Zynteglo), which treats transfusion-dependent beta thalassemia (TDT), a rare genetic blood disorder, and has been approved for use by the European Medicines ...
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CRISPR Therapeutics AG is a Swiss–American biotechnology company headquartered in Zug, Switzerland.It was one of the first companies formed to utilize the CRISPR gene editing platform to develop medicines for the treatment of various rare and common diseases.
Related: Verve Therapeutics Amends Gene Editing Pact With Beam Therapeutics. BMO says that the key poi. BMO Capital Markets has raised the price target on Beam Therapeutics Inc (NASDAQ: BEAM) to ...