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Session 3 Track 1: Extra Hepatic Delivery – Clinical Experience in CNS
Session Chair(s)
Barry Ticho, MD, PhD
Chief Medical Officer
Stoke Therapeutics, United States
Amy Kao
Medical Officer, Division of Neurology 2, OND, CDER
FDA, United States
Oligonucleotides are an emerging class of drugs with potential for the treatment of a wide range of central nervous system conditions. To date, there are two approved oligos for brain diseases and a large number of ongoing clinical trials. This session will review recent advances in chemical modifications and delivery techniques of oligonucleotides in clinical testing that are intended to enhance brain exposure and clinical efficacy.
Learning Objective : At the conclusion of this session, participants should be able to:- Compare various methods of delivery of oligos to the brain
- Understand the targets of new oligos for CNS diseases
- Evaluate the challenges and risks of clinical testing of oligos for brain diseases
Speaker(s)
Intrathecal Delivery of Antisense Oligonucleotides for Treatment of a Genetic Form of Epilepsy
Barry Ticho, MD, PhD
Stoke Therapeutics, United States
Chief Medical Officer
C16 Conjugated to siRNA to Target Cells in the Central Nervous System
Representative Invited
Alnylam Pharmaceuticals, United States
Transferrin Receptor-Mediated Brain Delivery of Enzymes and Oligonucleotides Using Transport Vehicle Technology
Representative Invited
Denali Therapeutics, United States
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