REGN5846

REGN5846 is an investigational gene therapy developed by Regeneron Pharmaceuticals for the treatment of hemophilia B, a genetic bleeding disorder caused by a deficiency in coagulation factor IX (FIX). The therapy utilizes an adeno-associated virus serotype 8 (AAV8) vector to deliver a functional copy of the human factor IX gene to hepatocytes in the liver. Once transduced, these liver cells produce and secrete the FIX protein into the bloodstream, potentially providing a long-term, endogenous source of the clotting factor and reducing or eliminating the need for regular prophylactic infusions of FIX concentrates. REGN5846 is currently being evaluated in early-stage clinical trials to assess its safety, tolerability, and efficacy in increasing FIX activity levels in patients with moderate-to-severe hemophilia B.

Other names

adeno-associated virus serotype 8 vector encoding human factor IXAAV8-hFIXAAV-8-hFIXAAV 8-hFIX