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DIA/FDA Oligonucleotide-Based Therapeutics Conference


Session 1: Rare Diseases

Session Chair(s)

Phil  Gatti, PhD

Phil Gatti, PhD

  • Pharmacologist
  • FDA, United States
Edward M. Kaye, MD

Edward M. Kaye, MD

  • Chief Executive Officer
  • Stoke Therapeutics, United States
Personalized medicine is a major goal in development of new drugs and in medical care in general. Unfortunately, this is very difficult considering the myriad of genetic variations and pathological mutations in people. Oligonucleotide treatments, however, allow for the targeted development of treatment in not only rare and ultrarare diseases, but recently for one person. In today’s Rare Diseases session, the speakers will demonstrate examples of this type of development program with its successes and challenges.

Speaker(s)

Timothy W. Yu, MD, PhD

Batten Disease Patient Story Update

Timothy W. Yu, MD, PhD

  • Assistant Professor, Harvard Medical School
  • Division of Genetics and Genomics, Boston Children’s Hospital, United States
Matt R. Buck, JD

Ultra-Rare Disease Development Scenario

Matt R. Buck, JD

  • Vice President, Regulatory Affairs
  • Ionis Pharmaceuticals, Inc., United States
Lucas  Kempf, MD

Ultra-Rare Disease Development Scenario

Lucas Kempf, MD

  • Medical Officer
  • FDA, United States