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Domofenogene zalfaparvovec

Development stage: Phase 2
Lead developer: BioMarin
Modality: Gene Therapies
Administration: Intravenous

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.

Targets

PAH (Phenylalanine hydroxylase)

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