GeneTherapy.ISTH.org - Gene Therapy Latest News

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

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

Human Gene Therapy for Hemophilia

Human Gene Therapy for Hemophilia

04 February 2020

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

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
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

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

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