Drug intelligence / Profile preview

GT0003X

Development stage
Unknown
Lead developer
ONODERA GT Pharma
Modality
AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Parenteral
01

Overview

**GT0003X** is an investigational, locally administered adeno-associated virus serotype 2 gene therapy developed by Onodera GT Pharma for moderate-to-advanced Parkinson's disease. It is a defined three-vector product comprising separate AAV2 vectors encoding **tyrosine hydroxylase**, **GTP cyclohydrolase 1**, and **aromatic L-amino acid decarboxylase**. Following bilateral intraputaminal infusion, the vectors are intended to restore striatal dopamine biosynthesis by enabling local production of L-DOPA, tetrahydrobiopterin cofactor, and dopamine, respectively. GT0003X is being evaluated in an ongoing first-in-human Phase 1/2 study in patients with Hoehn and Yahr stage IV Parkinson's disease.

02

Targets

TH (Tyrosine hydroxylase)GCH1 (GTP cyclohydrolase 1)DDC (Aromatic L-amino acid decarboxylase)

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