BSTP/STP Webinar
A Pathologist Perspective on the Non-Clinical Safety Assessment of GalNAc-conjugated RNAi Therapeutics
September 28, 2021
10:30 AM EDT, 3:30 PM GMT/ 4:30 PM CET
A BSTP webinar - in collaboration with the Society of Toxicological Pathology (STP) to be held on Tuesday 28th September 2021, at 10:30 AM Eastern Daylight Time (New York, GMT-04:00), 3:30 PM GMT Time (London, GMT+01:00), and the duration of the webinar will be up to 1 hour.
A Pathologist Perspective on the Non-Clinical Safety Assessment of GalNAc-conjugated RNAi Therapeutics
By Drs. Arlin Rogers and Wendell Davis (Alnylam Pharmaceuticals)
Abstract: The discovery and elucidation of the mechanism of RNA interference (RNAi) by Fire and Mello, and the subsequent work by Tuschi to demonstrate mammalian RNAi has spurred the field of small interfering RNA (siRNA)-based therapeutics for the treatment of a wide range of human disorders. siRNA-based therapeutics harness a highly conserved natural RNAi mechanism whereby short, 20-25 nucleotide-long, double-stranded RNAs catalyze the sequence-specific downregulation of a disease-causing messenger RNA (mRNA).
The field of siRNA-based therapeutics has recently matured with the approvals of ONPATTROTM and GIVLAARITM for the treatment of hereditary transthyretin-mediated amyloidosis and acute hepatic porphyria, respectively. Many other candidate siRNAs are currently in various stages of clinical development across several therapeutic areas.
In this webinar, we will discuss the science behind siRNA-based therapeutics, some features of a typical development package and the pathology findings frequently observed in toxicology studies. In addition, we will provide some insights into the process of identifying development candidates.
We look forward to your participation.