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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 encoding a full-length human methyl cytosine binding protein 2 (MECP2)
Date Designated: 09/06/2026
Orphan Designation: treatment of Rett syndrome
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
GeneTrek Therapeutics (Shanghai) Co., Ltd.
No. 536 Laolu Highway
Lingang New Area, China (Shanghai) Pilot Free Trade Zone
Shanghai, Shanghai 201308
China

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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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