Treatment of a Single Patient With CRD-TMH-001

• Status: Enrolling by invitation
• Trial end date: 2023-09-01
• Target enrollment: 0
• Participant gender: Male

Summary

The study is a single patient study intended to understand the effects of a gene-editing therapeutic to treat a rare mutation of Duchenne muscular dystrophy.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Cure Rare Disease, Inc
• Collaborator: University of Massachusetts, Worcester

Criteria

Inclusion Criteria:

• Completion of informed consent

• Confirmation of genetic mutation

• Confirmation of absence of elevated AAV9 NAbs

Exclusion Criteria:

• Any significant medical issue(s) (past or current) that would, in the opinion of the Principal Investigator (PI), prevent this patient from being dosed.