Gene Therapy Latest News | Experimental Gene Therapy Studies

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

30 October 2019

Liver Gene Therapy: Reliable and Durable?

28 October 2019

Advances and Challenges for Hemophilia Gene Therapy

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
Clinical Advances in Gene Therapy Updates on Clinical Trials of Gene Therapy in Haemophilia
12 August 2019
Therapeutic hFIX Activity Achieved After Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-existing Anti-AAV5 NABs
12 August 2019
The Impact of Pre-Existing Immunity on the Non-Clinical Pharmacodynamics of AAV5-Based Gene Therapy
12 August 2019

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

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