Paul Batty

Our educational review summarising the hepatic lifecycle of AAV gene therapy and how this is linked to treatment outcomes has been published in the Journal of Thrombosis and Haemostasis www.jthjournal.org/article/S153... #GeneTherapy #Hemophilia

Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B | NEJM Adeno-associated virus (AAV)–mediated gene therapy has emerged as a promising treatment for hemophilia B. Data on safety and durability from 13 years of follow-up in a cohort of patients who had be...

Important new publication of long term follow up following AAV Gene Therapy for Severe Hemophilia B. Durable Factor IX expression at 13 years with no late onset safety concerns. www.nejm.org/doi/full/10....

UKHCDO gene therapy taskforce: Guidance for implementation of haemophilia gene therapy into routine clinical practice for adults Introduction 2022 was a landmark year with two adeno-associated viral vectors (AAVs) receiving conditional marketing authorization from EMA for the treatment of persons with severe haemophilia A and.....

Really exciting to have gene therapy for haemophilia B as an option in the UK. It needed us to establish new models of care to ensure access and safety. UK guidance for implementation of gene therapy just published: doi.org/10.1111/hae....