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Session 4 Track 1: Oligonucleotide Therapies for Brain and Eye Diseases: Learnings From Recent Clinical Trials
Session Chair(s)
Barry Ticho, MD, PhD
Chief Medical Officer
Stoke Therapeutics, United States
Paul C. Brown, PhD
ODE Associate Director for Pharmacology and Toxicology, OND, CDER
FDA, United States
Speakers will showcase clinical programs representing brain and eye diseases at various stages of development using intrathecal or intravitreal delivery. Clinical efficacy as well as safety profile of these delivery approaches will be covered. The goal of the session is to update on clinical progress, highlight the advantages of oligo therapeutics for these related organs, and discuss potential issues related to oligo delivery.
Learning Objective : At the conclusion of this session, participants should be able to:
- Evaluate oligonucleotide therapies in development for CNS and ocular diseases
- Appraise results of recent clinical trials using oligonucleotides for treatment of eye and brain diseases
- Identify advantages and disadvantages of intrathecal and intravitreal delivery of oligos
Speaker(s)
Antisense Oligonucleotides for treatment of Genetic Epilepsy and Optic Atrophy: Barry Ticho, MD, PhD
Chief Medical Officer
Stoke Therapeutics, United States
Antisense Oligonucleotides for treatment of Neuromuscular Diseases: Toby Ferguson, MD, PhD
Vice President and Head Neuromuscular Development Unit
Biogen, United States
Antisense Oligonucleotides for Inherited Retinal Diseases: Aniz GIRACH Girach, MD
Chief Medical Officer
ProQR Therapeutics, United Kingdom
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