Director of Clinical In Vivo Gene TherapyDivision of Hematology, Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania, United States
OC 02.4 - Long-Term Follow-Up of Participants in the Phase I/II Trial of Dirloctocogene Samoparvovec (SPK-8011): Durable FVIII Expression and Clinically Meaningful Reduction of Bleeding
Saturday, June 22, 202413:45 – 14:00 ICT
PB0235 - Trial-in-progress: A two-part open-label study of an in vivo CRISPR/Cas9-based coagulation factor IX targeted gene insertion therapy (REGV131-LNP1265), in participants with hemophilia B (BEYOND-9)
Sunday, June 23, 202413:45 – 14:45 ICT
