Jeffrey Chamberlain, PhD

Title: Moderators

Bio: Dr. Jeffrey Chamberlain is a professor in the Departments of Neurology, Medicine, and Biochemistry, the McCaw Endowed Chair in Muscular Dystrophy at the University of Washington School of Medicine, and Director of the Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center of Seattle. The main focus of our research is on the muscular dystrophies with two major goals: to develop a better understanding of the molecular basis of the pathophysiology of the diseases, and to develop gene and cell therapies that will correct and treat the muscular dystrophies. Major targets for therapy include Duchenne muscular dystrophy and LGMD2I. Currently, the laboratory includes ~15 scientists conducting cutting edge studies that we hope to translate into clinical trials in the near future. We are working with some of the best doctors and scientists at Children's Hospital, Fred Hutchinson Cancer Research Center, University of Rochester, and Harborview Medical Center to most effectively tackle the challenge ahead: to find a treatment for muscular dystrophy.

Gene Therapy and Gene Editing Symposium

A brief overview of the strategy guiding efforts in gene therapy and gene editing, as well as critical updates from the companies in this space. Moderated discussion included.

Jeffrey Chamberlain, PhD

Moderators

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Reed Clark, PhD

Chief Science Officer, Ultragenyx Gene Therapy

Ultragenyx

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Olivier Danos, PhD

Chief Scientific Officer

REGENXBIO

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Jennifer Hodge, PhD

Team Lead, Rare Neurology USMA

Pfizer

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Alison Mcvie-Wylie, PhD

Vice President, Disease Area Executive, DMD

Vertex Pharmaceutical

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Carl Morris, PhD

Chief Scientific Officer

Solid Biosciences

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Deanna Tucker, PharmD

Senior Medical Science Liaison

Sarepta Therapeutics

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