Huntington's Treatment Quest Reaches First Pluripotent Stem Cell Trial, with Safety Now Under Review

First clinical trial evaluates stem cell therapy for Huntington's disease

An update on the first clinical trial evaluating a pluripotent stem cell-derived neural stem cell therapy for Huntington's disease was presented today at the ISSCR 2026 Annual Meeting.

Huntington's treatment quest reaches first pluripotent stem cell trial, with safety now under review

An update on the first clinical trial evaluating a pluripotent stem cell-derived neural stem cell therapy for Huntington's disease was presented at the ISSCR 2026 Annual Meeting. The Phase Ib/IIa REGEN4HD study represents a significant translational milestone following years of preclinical research, pivotal safety studies, regulatory engagement and clinical trial development.

First Clinical Trial of Stem Cell Therapy for Huntington’s Disease

A groundbreaking clinical trial has commenced, evaluating a pluripotent stem cell-derived neural stem cell therapy for Huntington’s disease (HD), marking a pivotal advance in regenerative medicine. This Phase 1b/2a study, known as REGEN4HD, was highlighted today at the ISSCR 2026 Annual Meeting, signaling the first-ever human trial of its kind targeting this devastating neurodegenerative disorder. […]

First Clinical Trial of Stem Cell Therapy for Huntington's Disease

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Pluripotent stem cell trial for Huntington's disease

Early-stage study marks milestone for regenerative medicine