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Bethesda North Marriott Hotel and Conference Center

Oct 28, 2019 7:00 AM - Oct 30, 2019 12:30 PM

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

DIA/FDA Oligonucleotide-Based Therapeutics Conference

Session 1: Rare Diseases

Session Chair(s)

Philip  Gatti, PhD

Philip Gatti, PhD


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.


Timothy W. Yu, MD, PhD

Batten Disease Patient Story Update

Timothy W. Yu, MD, PhD

Boston Children’s Hospital, United States

Associate Professor, Harvard Medical School; Division of Genetics and Genomics

Matt R. Buck, JD

Ultra-Rare Disease Development Scenario

Matt R. Buck, JD

Ionis Pharmaceuticals, Inc., United States

Vice President, Regulatory Affairs

Lucas  Kempf, MD

Ultra-Rare Disease Development Scenario

Lucas Kempf, MD

Parexel, United States

Vice President, RCS

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