GeneTherapy.ISTH.org - Gene Therapy Latest News

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 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

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

I agree with the Terms and conditions and the Privacy policy

About

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

Essential SSL