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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals

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Generic Name:  an antibody oligonucleotide conjugate (AOC), comprised of a human transferrin receptor 1 (TfR1) targeting, effector function null, humanized IgG1 antibody (AV01mAb) conjugated to one small interfering RNA (siRNA).
Date Designated:  07/21/2021
Orphan Designation:  Treatment of Myotonic Dystrophy Type 1 (DM1)
Orphan Designation Status:  Designated
FDA Orphan Approval Status:  Not FDA Approved for Orphan Indication
Avidity Biosciences
10975 N. Torrey Pines Road, Ste. 150
La Jolla, California 92037
United States

The sponsor address listed is the last reported by the sponsor to OOPD.
*Exclusivity Protected Indications are shown for approvals from Jan. 1, 2013, to the present.
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