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Session 10 Track 3: CMC and Regulatory Experience for Novel Oligonucleotide Therapeutics
Session Chair(s)
Rima Patel-Goel, MS
Senior Director Regulatory CMC, AADi Biosciences, Inc., United States
Benjamin Stevens, MPH
Senior Director, Regulatory Affairs CMC Policy and Advocacy, Alnylam Pharmaceuticals, Inc., United States
Several oligonucleotide classes (e.g., siRNAs, ASOs) have recently matured to validated therapeutic approaches with demonstrated utility across several indications. While industry and regulatory authorities have worked together to establish pathways for commercialization of these leading oligonucleotide classes, novel modalities present unique challenges to CMC development and broad regulatory acceptability. This session will include industry presentations representing two distinct novel oligonucleotide therapeutic modalities and their perspectives on CMC development challenges and experience with regulatory acceptability. The presentations will be followed by a panel discussion with representatives from both industry and regulatory authorities.
Learning Objective : At the conclusion of this session, participants should be able to:
- Formulate approaches for early stage CMC and regulatory development of novel oligonucleotide therapeutics
- Plan strategic interactions with regulators to ensure acceptability of CMC development approaches for novel oligonucleotide therapeutics
- Identify risks associated with CMC and regulatory development of novel oligonucleotide therapeutics
Speaker(s)
Kristy Wood, PhD
Vice President Nucleic Acid Therapeutics Development & Manufacturing, Intellia Therapeutics, United States
Speaker
Rachel Johns, PhD
Senior Director, Analytics and Formulations, Avidity Biosciences, Inc., United States
Antibody Oligonucleotide Therapeutics – from Development to the Clinic
Adam Dinerman, PhD
Executive Director, Head of Chemistry, Manufacturing and Controls (CMC), Aro Biotherapeutics, United States
Panelists
Martin Nemec, PhD
Senior Biologist/Evaluator, Health Canada, Canada
Panelists
Olen M. Stephens, PhD
Chemist Reviewer, CMC Reviewer, CDER/OPQ/ONDP, FDA, United States
Panelists
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