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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: a genetically modified AAV9 carrying an expression cassette that expresses human tripartite motif containing 72 (TRIM72) protein
Date Designated: 06/24/2025
Orphan Designation: treatment of amyotrophic lateral sclerosis
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
SineuGene Therapeutics Co., Ltd.
1st Floor, Building 15th, PKU Care Innovation Park, Life Park Road
Changping District
Beijing
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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