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devafidugene civaparvovec
- Development stage
- Unknown
- Lead developer
- Sangamo Therapeutics
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, Zinc Finger Nucleases → Other Nucleases → Programmable Nucleases → Gene Editing → Gene Therapies
- Administration
- Intravenous
01
Overview
Devafidugene civaparvovec is an investigational in vivo genome editing gene therapy developed by Sangamo Therapeutics. It utilizes proprietary zinc finger nucleases (ZFNs) delivered via an adeno-associated virus (AAV) vector to insert a functional copy of the alpha-L-iduronidase (IDUA) gene into patient cells. The primary aim is to restore IDUA enzyme activity for the treatment of mucopolysaccharidosis type I (MPS I), a rare lysosomal storage disorder caused by IDUA deficiency. The therapy has also been investigated for other indications, including hemophilia B and mucopolysaccharidosis type II, though its main focus remains MPS I[1][2][3].
- Other names
- devafidugene civaparvovec
02
Targets
ALB (Human serum albumin)
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