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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: adeno-associated viral vector serotype 9 containing the human PLA2G6 gene
Date Designated: 09/15/2023
Orphan Designation: Treatment of PLA2G6-associated neurodegeneration
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Bloomsbury Genetic Therapies Ltd.
30 Broad Street
Great Cambourne
Cambridge CB23 6HJ
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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