Testing a new gene therapy approach for severe bleeding disorder

This is an early-stage trial testing a new gene therapy called ANB-002 (arvenacogene sanparvovec) in men with hemophilia B, a genetic bleeding disorder where the body cannot make enough clotting factor. The study will give patients a single infusion of the medication at different dose levels to see if it's safe and whether it helps their bodies produce more clotting factor naturally. Researchers will carefully monitor each patient for safety before moving to the next dose level.

Hemophilia B patients currently need regular infusions of clotting factor throughout their lives to prevent dangerous bleeding. This trial explores whether a one-time gene therapy treatment could help the body make its own clotting factor, potentially reducing or eliminating the need for lifelong infusions.