English

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

Reed Clark, PhD

Chief Science Officer, Ultragenyx Gene Therapy

Ultragenyx

Olivier Danos, PhD

Chief Scientific Officer

REGENXBIO

Jennifer Hodge, PhD

Team Lead, Rare Neurology USMA

Pfizer

Alison Mcvie-Wylie, PhD

Vice President, Disease Area Executive, DMD

Vertex Pharmaceutical

Carl Morris, PhD

Chief Scientific Officer

Solid Biosciences

Deanna Tucker, PharmD

Senior Medical Science Liaison

Sarepta Therapeutics

Linking the Community to a Cure

CureDuchenne Link is a centralized data hub which combines clinical data, biosamples, and patient reported data for individuals with Duchenne and Becker muscular dystrophy, as well as carriers. Information and samples provided by participants are coded and available for use by approved researchers across the world.

Ana Christensen, MPH

Lead Project Manager, Strategic Solutions

TRiNDS