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lanacogene vosiparvovec

Development stage: Preclinical
Lead developer: Pfizer
Modality: Gene Therapies
Administration: Intravenous

Overview

Lanacogene vosiparvovec is an investigational gene therapy designed for the treatment of hemophilia B (congenital Factor IX deficiency). It utilizes an adeno-associated virus (AAV) vector to deliver a functional copy of the Coagulation factor IX gene to liver cells, enabling endogenous production of Factor IX protein and thereby reducing or eliminating the need for exogenous factor replacement therapy. The primary indication is hemophilia B, and it is being developed as a one-time intravenous infusion intended to provide long-term correction of the bleeding disorder[1][3]. The developer associated with this drug is Pfizer.

Other names: lanacogene vosiparvovec

Targets

F9 (Coagulation Factor IX)

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