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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, replication incompetent adeno-associated virus (AAV) viral vector platform with a serotype 8 capsid with a complementary DNA (cDNA) of the human GUCY2D gene encoding RetGC (AAV8-RK-RetGC)
Date Designated: 02/11/2026
Orphan Designation: treatment of inherited retinal dystrophy due to GUCY2D gene mutations
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
MeiraGTx UK II Ltd
92 Britannia Walk
London N1 7NQ
United Kingdom

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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.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