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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals

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Generic Name: Virus-like particle containing Cas9/gRNA ribonucleoprotein targeting the human HTT gene
Date Designated: 10/05/2023
Orphan Designation: Treatment of Huntington’s Disease
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Shanghai BDgene Co., Ltd.
301, 302, 303, 304, Unit B, Building 17
No.1699 Duhui Road
Minhang District, Shanghai 201108
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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