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AOC 1001, Avidity Biosciences lead product candidate utilizing its Antibody Oligonucleotide Conjugates (AOC) platform, is designed to address the root cause of myotonic dystrophy type 1 (DM1) by reducing levels of disease-related mRNA called DMPK.
Key Takeaways. An administrative order-on-consent (AOC) is an agreement between an individual, business, or other entity and a regulatory body in which they agree to pay for damages caused by violations and to cease the activities that caused the damages to occur.
AOC 1001, an RNA therapeutic designed to target the pathogenic driver of DM1, is a DMPK siRNA conjugated to a humanized antibody targeting human transferrin receptor 1 (TfR1). The antibody targets muscles for delivery of siRNA into the cytoplasm and nucleus where it mediates DMPK mRNA degradation.
A few months after the data, the FDA granted breakthrough therapy designation to AOC 1001 as a potential treatment for DM1. To date, it has also received orphan drug and fast track designations by both the FDA and European Medicines Agency.
In general, DM2 is a less severe disease than classic DM1. However, it may affect walking ability earlier than DM1 because it causes early weakening of the hip muscles. DM2 is rare compared to DM1, except in people of German descent.
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