Avidity Biosciences presents preclinical data from FSHD program

[Administrator]

June 17, 2022 – Avidity Biosciences, Inc., a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), joins in activities to raise awareness for Facioscapulohumeral muscular dystrophy (FSHD) in support of World FSHD Day and highlights preclinical results supporting AOC 1020 for the treatment of FSHD at the 29th Annual FSHD Society International Research Congress (FSHD IRC) in Orlando, Florida. Currently, there are no approved therapies for the treatment of FSHD.

"We are grateful for our partnership with the global FSHD community as we collectively work together toward developing treatments for one of the most common forms of muscular dystrophy. We continue to work closely with the community to advance AOC 1020 for the treatment of FSHD into the clinic by the end of this year," said Sarah Boyce, president and chief executive officer.

"Our preclinical data, presented at FSHD IRC, support our approach of directly targeting DUX4 with AOC 1020. The data demonstrate that a murine version of AOC 1020 prevents muscle weakness by blocking expression of DUX4 in a FSHD mouse model of disease. These findings are particularly important for progressive diseases like FSHD where aberrant and sporadic expression of DUX4 leads to cumulative damage across different muscle groups," said W. Michael Flanagan, Ph.D., Avidity's chief technical officer.

Today, Avidity will share preclinical data from the FSHD program during a presentation at the FSHD IRC. Studies were conducted in mouse models of FSHD that are genetically engineered to express human DUX4 in skeletal muscle. AOC 1020 is comprised of a DUX4-targeting siRNA conjugated to the human anti-transferrin receptor 1 monoclonal antibody. The preclinical studies were done using a monoclonal antibody suitable for mice. The preclinical data provides support for AOC 1020 to enter the clinic for the treatment of FSHD by end of 2022.

Avidity cautions readers that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on the company's current beliefs and expectations.

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https://www.aviditybiosciences.com/