Clinical Trial Update: Safety and Efficacy of scAAV9/AGA Gene Therapy in Participants With Aspartylglucosaminuria (AGU) [Phase 1, Phase 2]

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Summary

Phase 1/2 clinical trial evaluating scAAV9/AGA gene therapy for Aspartylglucosaminuria, with recruitment not yet started.

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Diseases / indications

• Aspartylglucosaminuria

Drugs mentioned

• scAAV9/AGA gene therapy (scAAV9-AGA)

Snippet

Sponsor: Rare Trait Hope | Conditions: Aspartylglucosaminuria, Aspartylglucosamidase (AGA) Deficiency | Phase 1, Phase 2 | Status: NOT_YET_RECRUITING