Drug intelligence / Profile preview

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)

Beyond the preview

Go deeper on GT0008X.

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Clinical trials

Follow clinical development from study design and recruitment through results.

Trial phase
Status
Readouts

Indications & development

Explore development by indication, patient population, and geography.

Indications
Development status
Countries

Licensing & deals

Trace asset ownership, licensing agreements, and commercial partnerships.

Partners
Deal terms
Milestones

Patents & exclusivity

Explore the patent landscape and regulatory exclusivity around an asset.

Patents
Expiration dates
Exclusivity

Competitive landscape

Compare development programs by target, modality, and indication.

Competing assets
Targets
Development stage

Research & analysis

Connect source evidence and development news to your research questions.

Publications
News
Analysis

Bring the full picture into focus.

See how Gosset can support your research on GT0008X.

Explore the full profile