GeneTherapy.ISTH.org - Gene Therapy Latest News

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

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

18 March 2020

High Dose of Gene Therapy for Hemophilia B Normalizing FIX Levels, Early Trial Data Show

High Dose of Gene Therapy for Hemophilia B Normalizing FIX Levels, Early Trial Data Show

18 February 2020

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

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

17 March 2020

Human Gene Therapy for Hemophilia

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
BioMarin submits EMA filing for first hemophilia gene therapy
18 December 2019
Enhanced Factor IX Activity following Administration of AAV5-R338L "Padua" Factor IX versus AAV5 WT Human Factor IX in NHPs
17 November 2019

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

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