戻る Agenda
Session 2 Track 1: The Brains Behind Delivery of Oligos to the CNS
Session Chair(s)
Barry Ticho, MD
Chief Medical Officer, Stoke Therapeutics, United States
Amy Kao, MD
Senior Physician, Division of Neurology 1 and 2, OND, CDER, 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.
Investigating Disease-Modifying Potential of Antisense Oligonucleotide
Puja Joshi, Stoke Therapeutics
The Brains Behind Delivery of Oligos to the CNS
Toby Ferguson, Alnylam Pharmaceuticals
Roshni Ramachandran, Denali Therapeutics
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)
Investigating Disease-Modifying Potential of Antisense Oligonucleotide
Puja Joshi, MD
Senior Medical Director - Clinical Development, Stoke Therapeutics, United States
The Brains Behind Delivery of Oligos to the CNS
Toby Ferguson, MD
Senior Vice President Neuroscience Therapeutic Area, Alnylam Pharmaceuticals, United States
Accelerating the Discovery and Development of Medicines for Neurodegeneration
Roshni Ramachandran, PhD
Director, Toxicology, Denali Therapeutics, United States