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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 gene editing therapy that uses a CRISPR (clustered regularly interspaced short palindromic repeats)-based adenosine base editor (ABE), delivered using a smooth muscle cell targeting adeno-associated virus (AAV) capsid called AAV-PR (comprised of the parent AAV9 capsid modified with a unique 7-mer amino acid after amino acid 588 of VP1 protein)
Date Designated: 09/26/2024
Orphan Designation: treatment of multisystemic smooth muscle dysfunction syndrome (MSMDS) caused by ACTA2-R179H mutation
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Mass General Brigham
65 Landsdowne St, Suite #142
Gene and Cell Therapy Institute
Cambridge, Massachusetts 02139
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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