Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides.

ABSTRACT: The nucleic acid therapeutics field has made tremendous progress in the past decades. Continuous advances in chemistry and design have led to many successful clinical applications, eliciting even more interest from researchers including both academic groups and drug development companies. Many preclinical studies in the field focus on improving the delivery of antisense oligonucleotide drugs (ONDs) and/or assessing their efficacy in target tissues, often neglecting the evaluation of toxicity, at least in early phases of development. A series of consensus recommendations regarding regulatory considerations and expectations have been generated by the Oligonucleotide Safety Working Group and the Japanese Research Working Group for the International Council for Harmonisation of Technical Requi

SUBMITTER: Goyenvalle A 

PROVIDER: S-EPMC9940817 | biostudies-literature | 2023 Jan

REPOSITORIES: biostudies-literature