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Session 6 Track 1: Safety Observations in Late Clinic Development and Early Commercial Surveillance
Session Chair(s)
Louis O'Dea, MD
Chief Medical Officer and President
BIORCHESTRA (USA) Inc, United States
Representative Invited
FDA, United States
Oligonucleotide therapeutics have become mainstream tools for regulation of rare and genetic diseases through their ability to modulate gene expression across a variety of processes. Generational advances in chemistry, delivery and targeting have positively impacted their safety profile. While challenges remain, cell-specific targeting has significantly decreased effective dose and reduced off-target effects, leading to improved safety. This session will address some of the strategies that have contributed to these improvements in safety.
Learning Objective : At the conclusion of this session, participants should be able to:- Understand the underlying advances that have contributed to the Safety of oligonucleotide-based therapeutics
- Understand the impact of specific targeting on exposure reduction and resulting Safety
- Hear about the expansion of oligonucleotide-based therapeutics to broader disease populations as a result of their improved benefit-risk profile
Speaker(s)
The Evolution of Safety in Oligonucleotide-based Therapeutics; the Roles of Chemistry, Formulation and Targeting
Representative Invited
Ionis Pharmaceuticals, Inc., United States
Clinical Case Study in Oligo Safety, TTR Amyloidosis Comparative Safety of Systemic vs Targeted Oligonucleotide Therapeutics
Representative Invited
Alnylam Pharmaceuticals, United States
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