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DIA/FDA Oligonucleotide-Based Therapeutics Conference

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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

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

Speaker(s)

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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