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For more companies, take a look at 5 Most Promising Gene Therapy Companies to Watch. Gene therapy is a revolutionary new technology that has followed the growing advances in computing power. The ...
Alipogene tiparvovec (Glybera): AAV-based treatment for lipoprotein lipase deficiency (no longer commercially available); Axicabtagene ciloleucel (Yescarta): treatment for large B-cell lymphoma [1]
Ultragenyx is based in Novato, CA and Brisbane, CA [2] and has a presence in the Boston area, including a gene therapy plant under construction as of 2021. [3] The company’s Latin American headquarters is located in Miami. [4]
Sangamo Therapeutics, Inc. (previously known as Sangamo Biosciences, Inc.) is an American biotechnology company based in Brisbane, California. It applies cell and gene therapy to combat haemophilia and other genetic diseases. [2]
Thanks to advances in technology and gene sequencing, gene therapy has gone from science fiction to science fact. By either adding new genes to fight faulty ones, replacing/editing missing/broken ...
Adverum Biotechnologies, Inc., formerly known as Avalanche Biotechnologies, [2] is a publicly traded [3] clinical stage gene therapy company located in Redwood City, California. [4] The company is targeting unmet medical needs for serious ocular and rare diseases, including wet age-related macular degeneration (wet AMD).
The following table lists the largest biotechnology and pharmaceutical companies ranked by market capitalization in billion US dollars. The change column indicates the company's relative position in this list compared to their relative position in the preceding year; i.e., an increase would be moving closer to rank 1 and vice versa.
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 ...
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