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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 rAAV8 capsid containing a single-stranded DNA template encoding wild-type human coagulation factor IX protein.LNP1265: a lipid nanoparticle encapsulating a Cas9 mRNA and a single guide RNA
Date Designated: 12/20/2023
Orphan Designation: Treatment of Hemophilia B
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Regeneron Pharmaceuticals Inc
777 Old Saw Mill River Road
Tarrytown, New York 10591
United States

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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.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