KL001 (Kanglin Biotechnology)

KL001 is an adeno-associated virus (AAV)-based gene therapy developed by Kanglin Biotechnology for the treatment of hemophilia B. It is designed to deliver a functional copy of the human coagulation factor IX (FIX) gene to the liver, enabling the endogenous production of FIX protein in patients with moderately severe to severe deficiency (residual FIX levels ≤2 IU/dl). By restoring FIX activity, the therapy aims to reduce or eliminate the need for exogenous factor replacement and prevent spontaneous bleeding episodes. KL001 is currently being evaluated in a dose-escalation pilot study at the Affiliated Hospital of Guangdong Medical University to assess its safety and efficacy.

Other names

KL001 injection solutionKL-001 injection solutionKL 001 injection solution