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Session 9 Track 2: From Data to Decisions: Evolving the Regulatory Landscape for Oligonucleotide Therapeutics
Session Chair(s)
Xuan Chi, DRSc, MD
Supervisory Pharmacologist, CDER, FDA, United States
Ronald Wange, PhD
Principal Consultant, Aclairo Pharmaceutical Development Group, Inc., United States
Presentations in this session will review general toxicology and carcinogenicity data gathered and analyzed by regulatory agencies and industry for oligonucleotide therapeutics with well-characterized mechanisms and compositions. The aim is to inform and enhance regulatory decision-making by applying risk-based approaches that build on existing knowledge and determine best practices for evaluating new chemistries.
Learning Objective : At the conclusion of this session, participants should be able to: - Understand the principles and examples of applying knowledge from “well characterized ONTs”, and discuss any concerns or challenges of this approach
- Identify data gaps for “well characterized ONTs” for certain endpoints
- Suggest innovative approaches to address the data gaps or challenges discussed above
Speaker(s)
Comparative Analysis of Chronic vs. Sub-chronic Toxicity Study Findings with Well-characterized ONTs
Christine Siezen, PhD
Senior Nonclinical Assessor, Dutch Medicines Evaluation Board, Netherlands
FDA Experience with Carcinogenicity Assessment of Oligonucleotide-based Products
Elena Braithwaite, PhD
Toxicologist, FDA, United States
Carcinogenicity Assessment Strategies for ONTs: OSWG Perspectives and Key Considerations
Scott Henry, DrPH
Senior Vice President, Nonclinical Development, Ionis Pharmaceuticals, Inc., United States
Speaker
Takasumi Shimomoto, DVM, PhD
Associate Senior Scientist for Toxicology, Pharmaceuticals and Medical Devices Agency, Japan
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