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adlinacogene civaparvovec

Development stage: Discontinued
Lead developer: Sangamo Therapeutics
Modality: Viral Vectors → Gene Addition/Replacement → Gene Therapies, Recombinant Proteins and Enzymes
Administration: Intravenous

Overview

Adlinacogene civaparvovec is an investigational gene therapy developed for the treatment of hemophilia B. It utilizes a proprietary zinc finger nuclease (ZFN) genome editing technology to insert a functional copy of the F9 gene, which encodes coagulation Factor IX, into the DNA of liver cells. The therapy is delivered using a recombinant non-replicating adeno-associated virus type 2/6 (rAAV2/6) vector. By enabling liver cells to produce endogenous Factor IX, it aims to restore normal blood clotting in patients with hemophilia B[1][3]. The drug was developed by Sangamo Therapeutics.

Other names: Factor IX gene therapy - Sangamo

Targets

F9 (Coagulation Factor IX)

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