CEO of Irish Haemophilia Society,former President of WFH and EHC. Supporter of Kerry GAA. Interested in politics,history,advocacy, books. All opinions personal.
Nov 9 • 13 tweets • 3 min read
20 Snippet thread from @wfhemophilia Novel products and Gene Therapy (GT) summit: 1. Work ongoing to develop capsids which transduce LSEC cells which would be better for FVIII GT 2. Regeneron working on drugs to prevent or suppress AAV ab to allow re-dosing or dosing those 1/13
who are AAV+. Includes blocking CD40 cells, or a bi specific antibody linvoseltamab or combinations 3. Liver fibrosis in mice leads to reduced vector uptake, alters vector clearance and biodistribution 4. Liver steatosis leads to increased vector uptake and faster loss 2/13
