The Food and Drug Administration (FDA) has granted Fast Track designation to AOC 1001 for the treatment of myotonic dystrophy type 1 (DM1).

DM1 is a rare genetic disorder caused by a triplet-repeat in the myotonic dystrophy protein kinase (DMPK) gene, resulting in toxic gain of function mRNA. The disease primarily affects skeletal and cardiac muscle and is characterized by muscle weakness, difficulty breathing, and cognitive and behavioral impairment.

AOC 1001 consists of a proprietary monoclonal antibody that binds to transferrin receptor 1 conjugated with a small interfering RNA targeting DMPK mRNA. The Company believes AOC 1001 will be able to address the underlying cause of DM1 by reducing levels of DMPK mRNA in skeletal, cardiac, and smooth muscle.

The designation is supported by data from preclinical studies, which showed that AOC 1001 successfully delivered the small interfering RNA to muscle cells leading to dose dependent reductions in DMPK mRNA. The Company will be investigating the safety and tolerability of AOC 1001 in adults with DM1 in the phase 1/2 MARINA study ( Identifier: NCT05027269).

“DM1 is an underrecognized, progressive and often fatal disease with no therapeutic options,” said Sarah Boyce, president and CEO of Avidity. “Fast Track designation for AOC 1001 underscores this unmet need and allows us to expeditiously work with FDA to potentially deliver this first-in-class therapy to people living with DM1 as quickly as possible.”


FDA grants Fast Track Designation to AOC 1001 for the treatment of myotonic dystrophy type 1. News release. Avidity Biosciences, Inc. Accessed October 18, 2021.