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dalnacogene ponparvovec

Development stage: Phase 1
Lead developer: Belief BioMed
Modality: Gene Therapies
Administration: Intravenous

Overview

Dalnacogene ponparvovec (BBM-H901) is a liver-tropic adeno-associated viral (AAV) vector gene therapy carrying a cassette coding for hyperactive Padua factor IX (FIX) protein. It is designed to deliver a functional copy of the human factor IX gene to liver cells, enabling continuous production and secretion of coagulation factor IX into the bloodstream to promote blood clotting in patients with hemophilia B. Administered as a single intravenous infusion, it offers a 'one-time administration and long-term effectiveness' approach that can potentially eliminate the need for regular factor IX replacement therapy.

Targets

F9 (Coagulation Factor IX)

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