Drug intelligence / Profile preview

RGL-193

Development stage
Phase 1
Lead developer
Regenelead Therapies
Modality
Gene Therapies
Administration
Parenteral
01

Overview

RGL-193 is an adeno-associated virus (AAV)-based gene therapy developed by Shanghai Regenelead Therapies for the treatment of advanced Parkinson's disease. The therapy is designed to deliver the DOPA decarboxylase (DDC) gene directly into the putamen, which facilitates the conversion of levodopa into dopamine, thereby addressing the dopamine deficiency characteristic of the disease. Additionally, RGL-193 stimulates the expression of glial cell line-derived neurotrophic factor (GDNF) to provide neuroprotective support to surviving dopaminergic neurons. The drug is administered via a one-time stereotactic bilateral injection into the putamen and is currently undergoing Phase 1 clinical trials to evaluate its safety, tolerability, and preliminary efficacy in patients who have become less responsive to standard oral medications.

02

Targets

GDNF (Glial cell line-derived neurotrophic factor)DDC (Aromatic L-amino acid decarboxylase)

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