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Since the approval of Vitravene (for the treatment of cytomegalovirus retinitis) in 1998, antisense oligonucleotide (ASO) therapies have evolved into a prominent class of therapeutics. In addition to the seven drugs, based on such molecules, that are commercially available, around 160 candidates are under development. Examples of recently approved antisense therapeutics include (in reverse chronological order) Viltepso (duchenne muscular dystrophy, March 2020), Vyondys 53 (duchenne muscular dystrophy, December 2019) and Waylivra (hereditary transthyretin-mediated (hATTR) amyloidosis, May 2019).
Given their ability to target the root cause of diseases, at the protein expression level, these disease-modifying interventions have potential applications across a wide range of therapeutic areas (including but not limited to oncological disorders, neurodegenerative disorders, respiratory disorders, and even certain