Author: [AUTHOR] Published on 1/13/2020 2:21:00 PM
ASCPT Members-Only Webinar presented by the Biologics Community (BIO), Pharmacometrics & Pharmacokinetics Community (PMK), Systems Pharmacology Community (SP), and Biomarkers & Translational Tools (BTT) Communities
Title: Clinical Pharmacology Considerations in Development of siRNA Therapeutics: Case Study of Givosiran
Date and Time: Tuesday, April 21, 2020, 1:00 pm ET
Speakers: Sagar Agarwal, PhD and Varun Goel, PhD
Moderator: Sagar Agarwal, PhD
Description: Small interfering RNAs (siRNAs) are a promising type of RNA-based therapeutic oligonucleotide drugs that can inactivate several target RNA molecules in a sequence-specific manner. Since the discovery of RNA interference (RNAi), a significant progress has been made in the development of therapeutic siRNAs including approval of the first ever siRNA therapy. Givosiran is a FDA approved siRNA therapeutic for the treatment of acute hepatic porphyria. Givosiran which is administered subcutaneously utilizes a GalNAc conjugate technology to enable targeted delivery to liver, the site of action. This presentation will give an overview of the clinical pharmacology program of givosiran, that included characterization of the PK and PD of givosiran, the impact of extrinsic and intrinsic factors, as well as special populations. Modeling and simulation analysis that enabled dose selection will be discussed.
Registration Link: hhttps://zoom.us/webinar/register/WN_idlcGg4PRsOVGKc0H3A1Rw