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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: Human transferrin 1 receptor (TfR1) targeting humanized IgG 1 kappa fragment antibody conjugated to an antisense oligonucleotide (ASO)
Date Designated: 09/19/2023
Orphan Designation: Treatment of Myotonic Dystrophy Type I
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Dyne Therapeutics, Inc.
1560 Trapelo Road
Waltham, Massachusetts 02541
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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