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Bethesda North Marriott Hotel and Conference Center

Apr 25, 2022 7:00 AM - Apr 27, 2022 12:00 PM

5701 Marinelli Road, , North Bethesda, MD 20852 , USA

DIA/FDA Oligonucleotide-Based Therapeutics Conference

DIA Your Way! Join us live in-person or live from the comfort of your home/office. This event offers two learning avenues depending on your preference!

Session 4 Track 1: Oligonucleotide Therapies for Brain and Eye Diseases: Learnings From Recent Clinical Trials

Session Chair(s)

Barry  Ticho, MD, PhD

Barry Ticho, MD, PhD

Chief Medical Officer

Stoke Therapeutics, United States

Paul C. Brown, PhD

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


Barry  Ticho, MD, PhD

Antisense Oligonucleotides for treatment of Genetic Epilepsy and Optic Atrophy: Barry Ticho, MD, PhD

Chief Medical Officer

Stoke Therapeutics, United States

Toby  Ferguson, MD, PhD

Antisense Oligonucleotides for treatment of Neuromuscular Diseases: Toby Ferguson, MD, PhD

Vice President and Head Neuromuscular Development Unit

Biogen, United States

Aniz GIRACH Girach, MD

Antisense Oligonucleotides for Inherited Retinal Diseases: Aniz GIRACH Girach, MD

Chief Medical Officer

ProQR Therapeutics, United Kingdom

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