SC3: Examining the Safety and Toxicity of Nucleic Acid Therapeutics

MONDAY, OCTOBER 19 | 6:30 – 9:30 PM (DINNER SERVED)

ABOUT THIS COURSE:

Nucleic acid drugs continue to deliver on their promise to become a third therapeutic modality, in addition to small molecules and biologics. Several antisense oligonucleotide drugs have been on the market for some time, while the first RNAi approval was granted in 2018. In addition, numerous mRNA and CRISPR therapeutic programs have entered clinical stages. Despite the common “nucleic acid” component, the mechanisms of action and of non-specific effects differ for each of these drug types.

Instructors:

Jeffrey Foy, PhD, Senior Director, Toxicology, Dicerna Pharmaceuticals

Shawna Weis, PhD, Senior Director, Regulatory Toxicology, Wave Life Sciences

Xiao Shelley Hu, Senior Director, Head of DMPK and Clinical Pharmacology, Wave Life Sciences

6:30 Dinner to be Served

6:50 Introduction and Welcome from the Chair

Chair to be Announced 

6:55 Nonclinical Strategies for GalNAc-Conjugated siRNAs (a Dicerna Perspective) and an Overview of the Oligonucleotide Safety Working Group (OSWG)

Jeffrey Foy, PhD, Senior Director, Toxicology, Dicerna Pharmaceuticals

This presentation will review the nonclinical strategies and results for compounds developed using Dicerna’s GalXC RNAi technology. An overview of the activities of the Oligonucleotide Safety Working Group (OSWG) will also be provided.

7:35 Nonclinical Considerations for the Development of Oligonucleotide Therapeutics

Shawna Weis, PhD, Senior Director, Regulatory Toxicology, Wave Life Sciences

This presentation will review some of the common toxicities associated with oligonucleotide administration, discuss the regulatory expectations for the nonclinical safety evaluation of oligonucleotide therapeutics in the context of the applicable guidance documents, and present case studies based on recently approved products.

8:15 Coffee and Dessert

8:30 ADME and PK/PD Considerations for Pre-clinical Development of Antisense Oligonucleotides

Xiao Shelley Hu, Senior Director, Head of DMPK and Clinical Pharmacology, Wave Life Sciences

Antisense oligonucleotides are emerging class of medicines for unmet medical needs. The ADME and PK/PD entail specific considerations to support effective preclinical development. This talk will focus on these factors relevant to pre-clinical development of antisense oligonucleotides.

9:10 Interactive Discussion with the Attendees

9:30 End of Course