Avidity : Preclinical Results Support AOC 1020 For Facioscapulohumeral Muscular Dystrophy Treatment

(RTTNews) - Avidity Biosciences Inc. (RNA) said Friday that its preclinical data supported its approach of directly targeting DUX4 with Antibody Oligonucleotide Conjugates or AOC 1020. The data demonstrated that a murine version of AOC 1020 prevents muscle weakness by blocking expression of DUX4 in a Facioscapulohumeral muscular dystrophy or FSHD mouse model of disease. Currently, there are no approved therapies for the treatment of FSHD.

The company presented the data at the 29th Annual FSHD Society International Research Congress in Orlando, Florida.

The company said it continues to work closely with the community to advance AOC 1020 for the treatment of FSHD into the clinic by the end of this year.

Facioscapulohumeral muscular dystrophy is characterized by progressive and often asymmetric skeletal muscle loss that initially causes weakness in muscles in the face, shoulders, arms and trunk, and progresses to weakness in muscles in lower body. FSHD is an autosomal dominant genetic disease, meaning a single copy of the disease-associated gene, DUX4 (double homeobox 4), is enough to cause the disease.

The abnormal expression of DUX4 leads to a series of downstream events that result in skeletal muscle wasting and compromised muscle function, including an inability to lift arms for more than a few seconds, loss of ability to show facial expressions and serious speech impediments.