Drug Profile

Research programme: facioscapulohumeral muscular dystrophy therapy - Avidity Biosciences

Alternative Names: AOC-FSHD

Latest Information Update: 28 Jun 2024

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At a glance

Originator Avidity Biosciences
Class Drug conjugates; Immunoconjugates; Monoclonal antibodies; Oligonucleotides; Small interfering RNA
Mechanism of Action DUX4L1 protein expression inhibitors; RNA interference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity Yes

28 Jun 2024 No recent reports of development identified for research development in Facioscapulohumeral-muscular-dystrophy in USA (Parenteral)
15 Mar 2021 Avidity Biosciences plans a clinical trial in Facioscapulohumeral muscular dystrophy in 2022
08 Jan 2021 Avidity Biosciences announces intention to file an IND for Facioscapulohumeral muscular dystrophy in 2022

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