Oligonucleotide-Based Therapeutics Conference

Convening industry and health authorities to inform, educate, and share advancements in oligonucleotide-based therapeutic product development.

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Session 6 Track 1: Different Paths, Common Goals: Global Regulatory Perspectives on FIH Trials for Oligonucleotide Therapies

Session Chair(s)

Andrew Slugg, MBA, MS

Senior Vice President, Global Head of Regulatory Sciences

Alnylam Pharmaceuticals, United States

Applications for first-in-human (FiH)trials for oligonucleotide therapies present unique challenges, as sponsors must navigate evolving scientific considerations alongside differing regulatory expectations across regions. This session brings together senior leaders from global health agencies to share expectations on clinical trial and investigational new drug applications, assess risk, approach safety monitoring, guide engagement, and weigh platform understandings and prior knowledge in FIH applications. Through candid agency perspectives and cross-regional dialogue, attendees will gain practical insight into common application pitfalls and regulatory expectations. Participants will leave better equipped to design and strengthen FiH trial applications that are robust, aligned, and globally informed.

Learning Objective :
Compare global regulatory expectations for first-in-human oligonucleotide trials, including key application content, approaches to dose selection and escalation, and safety monitoring
Identify common deficiencies in FIH oligonucleotide applications and apply best practices to strengthen submissions across regions
Apply health agency perspectives on use of platform knowledge and early regulatory engagement to improve FIH trial design and amendments