GeneTherapy.ISTH.org - Gene Therapy Latest News

BioMarin Submits Biologics License Application to U.S. Food and Drug Administration for Valoctocogene Roxaparvovec to Treat Hemophilia A

BioMarin Submits Biologics License Application to U.S. Food and Drug Administration for Valoctocogene Roxaparvovec to Treat Hemophilia A

02 January 2020

BioMarin submits EMA filing for first hemophilia gene therapy

BioMarin submits EMA filing for first hemophilia gene therapy

18 December 2019

Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A

Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A

02 January 2020

Enhanced Factor IX Activity following Administration of AAV5-R338L "Padua" Factor IX versus AAV5 WT Human Factor IX in NHPs

Enhanced Factor IX Activity following Administration of AAV5-R338L "Padua" Factor IX versus AAV5 WT Human Factor IX in NHPs

17 November 2019

Patient perspectives on novel treatments in hemophilia A: a qualitative study
17 November 2019
uniQure Granted US Patent to Use FIX-Padua for Hemophilia B Gene Therapy
05 November 2019
Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B
30 October 2019
Advances and Challenges for Hemophilia Gene Therapy
28 October 2019

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

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