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Domofenogene zalfaparvovec
- Development stage
- Phase 2
- Lead developer
- BioMarin
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
Domofenogene zalfaparvovec is a gene therapy developed by BioMarin Pharmaceutical, Inc. It is designed to treat phenylketonuria (PKU), a rare genetic metabolic disorder. The drug's mechanism of action involves gene transference, where it acts as a phenylalanine hydroxylase (PAH) agonist to restore or enhance the body's ability to metabolize phenylalanine. It has received orphan drug designation for phenylketonuria.
02
Targets
PAH (Phenylalanine hydroxylase)
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