Drug intelligence / Profile preview

VGN-R09b

Development stage
Phase 2
Lead developer
Shanghai Vitalgen BioPharma
Modality
AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intraparenchymal
01

Overview

VGN-R09b is an adeno-associated virus (AAV)-based gene therapy developed for the treatment of Parkinson's disease and aromatic L-amino acid decarboxylase (AADC) deficiency. The therapy utilizes an AAV9 vector to deliver a functional copy of the DDC gene, which encodes aromatic L-amino acid decarboxylase, directly into the striatum via intraparenchymal injection. This approach aims to restore or enhance dopamine synthesis in patients with dopamine-related disorders by increasing AADC enzyme activity in targeted brain regions. The drug is currently in Phase 1/2 clinical trials for Parkinson's disease and early-phase trials for AADC deficiency[1][3][5][6].

02

Targets

DDC (Aromatic L-amino acid decarboxylase)

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