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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: AAV-mediated anti-PROX1 gene therapy (AAV2-anti-PROX1-scFvFc)
Date Designated: 09/23/2026
Orphan Designation: treatment of retinitis pigmentosa
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Celliaz Ltd.
Room 205, Jinri Hall, 193 Munji-ro,
Yuseong-gu, Deajeon 34051
South Korea

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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.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