Clinical Trial Results: Stem Cell Gene Therapy for Cystinosis [Phase 1, Phase 2]

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Summary

A Phase 1/2 clinical trial evaluating a stem cell gene therapy for cystinosis completed, with results posted.

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Diseases / indications

• Cystinosis

Snippet

Sponsor: University of California, San Diego | Conditions: Lysosomal Storage Diseases, Cystinosis | Phase 1, Phase 2 | Status: COMPLETED | Results posted: 2026-08-06