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HLX-0201
- Development stage
- Unknown
- Lead developer
- Healx
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
HLX-0201 is an investigational oral combination therapy being developed by Healx for the treatment of Angelman syndrome. It consists of three repurposed active ingredients: n-acetylcysteine (NAC), alpha-lipoic acid (ALA), and a liposomal formulation of glutathione. Identified through Healx's AI-driven drug repurposing platform, HealNet, the combination is designed to address the oxidative stress and mitochondrial dysfunction that occur as a result of UBE3A gene deficiency in Angelman syndrome. By enhancing cellular antioxidant capacity and reducing reactive oxygen species (ROS), HLX-0201 aims to mitigate neurodevelopmental deficits and improve overall neurological function. The therapy is currently being evaluated in Phase 2 clinical trials to assess its safety, tolerability, and efficacy in patients with the disorder.
- Other names
- n-acetylcysteine + alpha-lipoic acid + glutathioneNAC + ALA + GSH
02
Targets
GSH (Glutathione)PDH (Pyruvate dehydrogenase complex)
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