GeneTherapy.ISTH.org - Gene Therapy Latest News

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

uniQure Granted US Patent to Use FIX-Padua for Hemophilia B Gene Therapy

uniQure Granted US Patent to Use FIX-Padua for Hemophilia B Gene Therapy

05 November 2019

Patient perspectives on novel treatments in hemophilia A: a qualitative study

Patient perspectives on novel treatments in hemophilia A: a qualitative study

17 November 2019

Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B

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
Liver Gene Therapy: Reliable and Durable?
28 October 2019
Gene Therapy for Hemophilia: Progress to Date and Challenges Moving Forward
28 October 2019
Immunoadsorption enables successful rAAV5-mediated repeated hepatic gene delivery in nonhuman primates
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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