Drug Profile

VGM R02b

Alternative Names: rAAV9-GCDH; VGM-R02; VGM-R02b

Latest Information Update: 31 May 2024

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At a glance

Originator Shanghai Vitalgen BioPharma
Class Gene therapies
Mechanism of Action Gene transference; Oxidoreductase replacements

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

Yes - Glutaric acidemia I
New Molecular Entity No

Highest Development Phases

Phase I Glutaric acidemia I

Most Recent Events

29 Apr 2024 Phase-I clinical trials in Glutaric acidemia I (In children, In infants, In neonates, Treatment-experienced) in China (Intraventricular) (NCT06217861)
27 Apr 2023 Vitalgen plans a clinical trial of VGM R02b in Glutaric acidemia I in China
11 Jul 2022 Pharmacodynamics data from a preclinical trial in Glutaric acidemia I released by Vitalgen (Vitalgen website,July 2022)

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