Rett Syndrome Genetic Medicines Summit 2023

In 2023 we hosted the first meeting of its kind with biopharma companies front and center. The Summit gathered basic scientists, pre-clinical, clinical, and regulatory experts to foster discussion, refine expectations, and identify best practices to expedite genetic medicine development.

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Featured Speakers

Adrian Bird, PhD

Buchanan Chair of Genetics, University of Edinburgh

Stuart Cobb, PhD

University of Edinburgh, Chief Scientific Officer, Neurogene

David R. Liu, PhD

Richard Merkin Professor, Broad Institute of MIT and Harvard, HHM

Peter Marks, MD, PhD

Director, Center for Biologics Evaluation and Research, FDA

Sukumar Nagendran, MD

President and Head of R&D, Taysha Gene Therapies

Huda Zoghbi, MD

Baylor College of Medicine, HHMI, The Jan and Dan Duncan Neurological Research Institute

Summit Program Book

RSRT invited 130 scientists and clinicians from 20 academic institutions, 43 biopharmaceutical companies, NIH and FDA to participate at the Summit via presentations, panel discussions and poster session.

In Their Own Words

Peter Beal, PhD

Adrian Bird, PhD

Stuart Cobb, PhD

Cary Fu, MD

Dina Katabi, PhD

Eric Marsh, MD, PhD

Rachel McMinn, PhD

John Sinnamon, PhD

Fyodor Urnov, PhD

Event Sponsors

"RSRT has done an incredible job. The number of modalities and the caliber of intellect in the room is off the charts high. These are the top people in their fields and they're interested in Rett syndrome. That's not easy to do."

Rachel McMinn, PhD

CEO, Neurogene