Gene Therapy Latest News | Experimental Gene Therapy Studies

Phase 3 Trial of AMT-061 Gene Therapy for Hemophilia B Exceeds Target Dosing

27 March 2020

Gene & Anti-TFPI Therapies in Hemophilia: Competitive Grant Program

18 March 2020

Hemophilia Gene Therapy Knowledge and Perceptions: Results of an International Survey

19 March 2020

New Gene Therapy Shows Promise in Hemophilia A Mouse Model, Study Shows

17 March 2020

High Dose of Gene Therapy for Hemophilia B Normalizing FIX Levels, Early Trial Data Show
18 February 2020
Human Gene Therapy for Hemophilia
04 February 2020
BioMarin Submits Biologics License Application to U.S. Food and Drug Administration for Valoctocogene Roxaparvovec to Treat Hemophilia A
02 January 2020
Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A
02 January 2020

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Supported by educational grants from BioMarin, Freeline Therapeutics Limited, Pfizer Inc., Shire, Spark Therapeutics, and uniQure, Inc

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