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adlinacogene civaparvovec
- Development stage
- Discontinued
- Lead developer
- Sangamo Therapeutics
- Modality
- Recombinant Proteins and Enzymes, Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous
01
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
02
Targets
F9 (Coagulation Factor IX)
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