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CLINICAL_TRIALS trial_result · 2026-01-14

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

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Summary

The clinical trial update reports completion of the Phase 1/2 study evaluating stem cell gene therapy for cystinosis.

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Diseases / indications

Snippet

Sponsor: University of California, San Diego | Conditions: Lysosomal Storage Diseases, Cystinosis | Phase 1, Phase 2 | Status: COMPLETED