Sarah Boyce, President and CEO of Avidity Biosciences, gives an overview of facioscapulohumeral muscular dystrophy (FSHD) and the technology her company is developing as a potential treatment for the disease.
FSHD is a rare, progressive musculoskeletal disease in which persons initially lose muscle strength in the face, shoulders, arms, and trunk, but later the disease progresses throughout the lower body. FSHD is due to the mis-expression of DUX4 in skeletal muscles. There are currently no approved treatments for the disease.
As Ms. Boyce explains, Avidity is currently developing a new class of oligonucleotide therapies called antibody oligonucleotide conjugates (AOC) therapeutics which, according to the company, “combines the tissue selectivity of monoclonal antibodies and the precision of oligonucleotide therapies”. Ms. Boyce is hopeful that AOC therapeutics will lead to the development of the first ever approved treatment of FSHD and benefit other rare musculoskeletal diseases as well such as myotonic dystrophy type 1 and duchenne muscular dystrophy (DMD). The company is hoping to begin clinical trials for myotonic dystrophy type 1 later this year, FSHD next year, and DMD the following year.
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