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Coagulin-B
- Development stage
- Discontinued
- Lead developer
- MediciNova
- Modality
- Gene Therapies
- Administration
- Intra-arterial, Intramuscular
01
Overview
Coagulin-B is an early-generation gene therapy developed by Avigen for the treatment of severe hemophilia B, a genetic disorder caused by a deficiency in clotting factor IX. The therapy utilizes an adeno-associated virus serotype 2 (AAV2) vector to deliver a functional human coagulation factor IX (FIX) gene. Administered via infusion into the hepatic artery, the vector is designed to transduce hepatocytes, turning the liver into a factory for the endogenous production of clotting factor IX. While Coagulin-B was a pioneering candidate in the field of gene therapy, clinical trials revealed that while the procedure was safe, the expression of FIX was transient due to a cytotoxic T-cell response against the AAV capsid, which led to the clearance of transduced hepatocytes. This discovery was pivotal for the field but ultimately led to the discontinuation of the Coagulin-B program in favor of next-generation vectors and immunosuppression strategies.
- Brand names
- Coagulin-B
- Other names
- AAV-human Factor IX vectorAAV-hFIX1AAV-hFIX-1AAV-hFIX 1
02
Targets
F9 (Coagulation Factor IX)
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