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GT0002X

Development stage: Unknown
Lead developer: ONODERA GT Pharma
Modality: Gene Therapies
Administration: Parenteral

Overview

GT0002X is an adeno-associated virus (AAV) vector-based gene therapy developed by ONODERA GT Pharma. It is designed to deliver the gene encoding for aromatic L-amino acid decarboxylase (AADC), an enzyme essential for the synthesis of dopamine and serotonin. The therapy is primarily being developed for the treatment of AADC deficiency, a rare genetic disorder, and Parkinson's disease, where it aims to restore dopamine production in the striatum. The program is currently in Phase 1/2 clinical development for both indications, with some rights reportedly out-licensed.

Targets

DDC (Aromatic L-amino acid decarboxylase)

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