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GT0011X
- Development stage
- Preclinical
- Lead developer
- Gene Therapy Research Institute
- Modality
- Gene Therapies
- Administration
- Intrathecal
01
Overview
GT0011X is an investigational adeno-associated virus (AAV) gene therapy being developed by ONODERA GT Pharma for the treatment of intractable or refractory epilepsy. The therapy is designed to deliver a therapeutic transgene to the central nervous system to modulate neuronal activity, offering a potential long-term solution for patients who do not achieve adequate seizure control with conventional anti-epileptic medications. By utilizing AAV vector technology, GT0011X aims to address the underlying pathophysiology of severe epilepsy through targeted genetic intervention. As of 2024, the program is in the preclinical stage of development.
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