Drug Profile

Research programme: Pompe disease therapy - Avidity Bioscieneces

Alternative Names: AOC-Pompe disease

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 Glycogen synthase kinase modulators; 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 Glycogen-storage-disease-type-II in USA (Parenteral)
19 May 2020 Early research in Glycogen storage disease type II in USA (Parenteral) (Avidity Biosciences pipeline, May 2020)

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