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GT0008X

Development stage: Preclinical
Lead developer: ONODERA GT Pharma
Modality: Gene Therapies

Overview

GT0008X is an adeno-associated virus (AAV) vector-based gene therapy being developed by ONODERA GT Pharma for the treatment of ornithine transcarbamylase (OTC) deficiency. OTC deficiency is an X-linked genetic disorder characterized by a lack of the OTC enzyme, leading to the accumulation of toxic levels of ammonia in the blood (hyperammonemia). GT0008X aims to deliver a functional copy of the OTC gene to the liver, thereby restoring enzyme activity and preventing hyperammonemic crises. The program is currently in the preclinical stage of development.

Targets

OCT (Organic cation transporter family)

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