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Avidity’s Phase I/II Success In Myotonic Dystrophy Obscured By Caveats

Dose-Dependent Response Unclear

 
• By Ayisha Sharma

The US firm’s lead antibody oligonucleotide conjugate candidate has improved muscle strength and mobility in an early muscular dystrophy trial but some experts are disappointed by dose-specific data and a serious adverse event.  

Pair of outstretched hands held open
Video Hand Opening Time Is One Of The Measures Of Muscle Function • Source: Shutterstock

Avidity Biosciences, Inc.’s lead candidate, AOC 1001, has enhanced muscle function in the Phase I/II MARINA trial of patients with myotonic dystrophy type 1 (DM1), sparking plans for pivotal development even as some analysts criticized muddied dose-related data.

DM1 is an often fatal form of muscular dystrophy caused by a triplet-repeat in the DMPK gene which leads to a toxic gain of functional mRNA

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