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Avidity Biosciences Supports World Facioscapulohumeral Muscular Dystrophy (FSHD) Day and Presents Preclinical Data from FSHD Program
Oral Presentation at FSHD IRC highlights preclinical data that demonstrated prevention of muscle weakness by reducing DUX4 expression AOC 1020 on track to be
About this update from Atrium Therapeutics, Inc.
[{"type":"text","content":"Oral Presentation at FSHD IRC highlights preclinical data that demonstrated prevention of muscle weakness by reducing DUX4 expression\nAOC 1020 on track to be in the clinic by end of 2022 for the treatment of FSHD\nSAN DIEGO, June 17, 2022 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), 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.\n\n \n \n \n \n \n \n\n \n\"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.\nFSHD is a rare, hereditary muscle-weakening condition that affects approximately 16,000-38,000 people in the United States. It is marked by life-long, progressive loss of muscle function and causes significant pain, fatigue, and disability. FSHD is an autosomal dominant disease caused by the abnormal expression of DUX4 (double homeobox 4), a gene involved in embryonic development but not typically expressed in muscles. This abnormal expression of DUX4 leads to a series of downstream events that result in skeletal muscle wasting and compromised muscle function with onset often in teenage and early adult years. \n\"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.\nEvery June 20th, people around the wo...