Pharma Focus Asia

Avidity Biosciences Discovers Novel Delpacibart Etedesiran (AOC 1001) for Myotonic Dystrophy Type 1

Avidity Biosciences has pioneered a groundbreaking treatment, del-desiran (AOC 1001), for Myotonic Dystrophy Type 1 (DM1). This investigational therapy targets the root cause of DM1, a progressive neuromuscular disorder with no approved treatments.

Del-desiran, Avidity's leading candidate developed using its AOC platform, aims to reduce levels of DMPK mRNA, a key factor in DM1 pathology. It comprises a unique monoclonal antibody binding to transferrin receptor 1 (TfR1), combined with a siRNA targeting DMPK mRNA.

In preclinical trials, del-desiran effectively delivered siRNAs to various muscle types, resulting in substantial and lasting reductions of DMPK RNA levels. This includes skeletal, cardiac, and smooth muscles, offering broad therapeutic potential.

Currently in Phase 1/2 development, del-desiran has completed the MARINA® trial and continues with the MARINA-OLE™ trial involving adults with DM1. DM1, characterised by a triplet-repeat mutation in the DMPK gene, manifests in diverse symptoms, including muscle weakness, respiratory issues, cardiac abnormalities, and cognitive impairment.

DM1, stemming from a triplet-repeat mutation in the DMPK gene, presents a spectrum of symptoms, including muscle weakness, respiratory issues, cardiac abnormalities, and cognitive impairment. 

The U.S. food and drug administration (FDA) has granted breakthrough therapy designation to delpacibart etedesiran (AOC 1001) for the treatment of myotonic dystrophy type 1 (DM1).

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