LNP1265

LNP1265 is an investigational gene therapy component used in combination with REGV131 for the treatment of Hemophilia B. This therapy utilizes CRISPR/Cas9 technology to insert a functional Factor IX gene into the patient's DNA. The goal is to enable patients with severe or moderately severe Hemophilia B—caused by mutations in the coagulation Factor IX gene—to produce their own clotting factor IX protein long-term, potentially reducing or eliminating the need for regular factor replacement therapy. The treatment is administered as an intravenous infusion and aims to provide a durable solution by giving liver cells genetic instructions to synthesize functional clotting factor IX[1][2][3][6][8].

Other names

REGV131-LNP1265REGV-131-LNP1265REGV 131-LNP1265LNP-1265/REGV-131