Gene-Edited cells aim to stop bleeding in severe hemophilia b
Disease control
Stopped early
This early-stage trial tests a one-time treatment called BE-101 for adults with severe hemophilia B. The therapy uses a person's own blood cells, modified with CRISPR gene editing, to continuously produce the missing clotting factor. The goal is to reduce or prevent bleeding epis…
Phase 1/2 • Sponsor: Be Biopharma • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC