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Session 2 Track 1: The Brains Behind Delivery of Oligos to the CNS
Session Chair(s)
Barry Ticho, MD, PHD
Chief Medical Officer
Stoke Therapeutics, United States
Representative Invited
FDA, United States
The session will highlight the successes and challenges of delivering oligonucleotides to the brain, particularly overcoming the blood–brain barrier while achieving sufficient distribution and durability of effect in patients. Solution will focus on optimizing intrathecal dosing strategies, improving oligonucleotide chemistry for stability and potency, and exploring novel delivery platforms to enhance brain uptake. The discussion will emphasize balancing efficacy, safety, and patient burden to enable broader clinical adoption.
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 Zorevunersen Demonstrates Potential as a Disease-modifying Therapy in Patients with Dravet Syndrome
Barry Ticho, MD, PHD
Stoke Therapeutics, United States
Chief Medical Officer
Speaker
Representative Invited
Alnylam Pharmaceuticals, United States