Paul Batty
@paulbatty.bsky.social
Clinician Scientist interested in haemophilia and gene therapy. University College London
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
The Hepatic Odyssey of AAV: From Gene Delivery to Long-Term Outcomes
Adeno-associated virus (AAV) vectors are the most developed approach for in-vivo gene
therapy. This treatment has resulted in therapeutically meaningful treatment responses
for a range of monogenic in...
www.jthjournal.org
September 16, 2025 at 3:00 PM
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
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....
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...
www.nejm.org
June 12, 2025 at 7:42 AM
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....
Reposted by Paul Batty
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....
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.....
doi.org
November 20, 2024 at 8:41 PM
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....