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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: Recombinant serotype 9 adeno-associated virus (AAV) encoding an intron encoding a miRNA generating sequence, complementary miRNA binding sites and a full-length human methyl cytosine binding protein 2 (MECP2)
Date Designated: 02/27/2023
Orphan Designation: Treatment of Rett syndrome
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Neurogene Inc.
535 W 24th St, 5th Floor
New York, New York 10011
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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