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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: A recombinant adeno-associated virus serotype 9 (rAAV9) vector carrying a compact high-fidelity Cas12i (hfCas12Max) and a CRISPR RNA (crRNA) targeting human DMD exon 51 SD site
Date Designated: 01/04/2024
Orphan Designation: Treatment of Duchenne Muscular Dystrophy (DMD) patients amenable to exon 51 skipping
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Cholgene Therapeutics, Inc.
54 Old Highway 22, Suite 220,
Clinton, New Jersey 08809
United States

The sponsor address listed is the last reported by the sponsor to OOPD.
*Exclusivity Protected Indications are shown for approvals from 01/01/2013 to the present.
*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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