Adverse Drug Reactions and Toxicity of the Food and Drug Administration–Approved Antisense Oligonucleotide Drugs

• Published in: Drug metabolism and disposition Vol. 50; no. 6; pp. 879 - 887
• Main Authors: Alhamadani, Feryal, Zhang, Kristy, Parikh, Rajvi, Wu, Hangyu, Rasmussen, Theodore P., Bahal, Raman, Zhong, Xiao-bo, Manautou, José E.
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 01.06.2022; The American Society for Pharmacology and Experimental Therapeutics
• Subjects: Chemical and Drug Induced Liver Injury - drug therapy; Chemical and Drug Induced Liver Injury - etiology; Drug-Related Side Effects and Adverse Reactions; Humans; Oligonucleotides - adverse effects; Oligonucleotides, Antisense - adverse effects; Oligonucleotides, Antisense - genetics; Special Section on Pharmacokinetics and ADME of Biological Therapeutics; United States; United States Food and Drug Administration; United States; AST; FDA; SMA; VEGF; ALT; VOD; HoFH; CMV; ADR; TTR; ULN; AMD; DMD; ASO; REMS

The market for large molecule biologic drugs has grown rapidly, including antisense oligonucleotide (ASO) drugs. ASO drugs work as single-stranded synthetic oligonucleotides that reduce production or alter functions of disease-causing proteins through various mechanisms, such as mRNA degradation, ex...