Vice President of Preclinical Development Ionis Pharmaceuticals Carlsbad, California, United States
Oligonucleotide-based therapeutics, designed to treat a variety of local and systemic diseases, are novel and relatively unknown to the public. Over the past 30+ years, RNA-targeting drugs have evolved significantly, expanding their reach to an array of diseases. Several antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs) have been approved, helping patients overcome serious illnesses. The goals of this presentation are to provide an update on the current status of ASO platforms and to overview the transformation in emerging ASO chemistries and nonclinical safety assessments. Additionally, the presentation will discuss the design of effective nonclinical safety assessments for ASOs, including regulatory guideline information, appropriate species selection, and determination of safety margins.
