Early data for Avidity’s RNA therapy show possible functional benefit in muscular dystrophy

Avidity Biosciences’ AOC 1020, newly dubbed del-brax, demonstrated a safe profile and was tied to functional improvement among people with facioscapulohumeral muscular dystrophy (FSHD) in a phase 1/2 trial.

The initial data drop assesses del-brax, a novel RNA therapeutic Avidity has dubbed an “antibody oligonucleotide conjugate,” among people with FSHD. The rare genetic disease progressively weakens muscles and causes significant pain, fatigue and disability. Currently, no approved treatments for the condition exist.

The double-blind trial tested single and multiple doses of del-brax among 39 participants with FSHD. The therapy demonstrated a favorable safety and tolerability profile—the primary objective of the study—with no serious adverse events reported and zero discontinuations.

The investigational therapy, which is designed to treat the abnormal expression of a gene called double homeobox 4 (DUX4), was also tied to a greater than 50% mean reduction across multiple DUX4 regulated gene panels for 12 patients receiving 2…
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