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

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

Event Photos

Event Attendees

Monica Coenraads, CEO, RSRT

Huda Zoghbi

Adrian Bird

Thomas Carroll, neurosurgeon and father of a girl with Rett syndrome

Gaia Skibinski

David Liu

David Liu, Winston Yan, Jeremy Duffield, Peter Marks, Brett Staahl

Jana von Hehn, CSO, RSRT

Neurogene team: Rachel McMinn, Stuart Cobb, Albena Patroneva, Andrew Mulberg

Huda Zoghbi

Qin Yu

Peter Marks, Director CBER, FDA

Randy Carpenter, CMO, RSRT and Jana von Hehn, CSO, RSRT

Peter Beal

Frank Sasinowski (moderator), Fyodor Urnov, Eric Marsh, Liz Berry-Kravis, Peter Marks, Andrew Mulberg, PJ Brooks. Toby Ferguson

Sukumar Nagendran

Sasha Jacobs

Stuart Cobb

Seng Cheng (moderator), Guoping Feng, Sukumar Nagendran, Liz Berry-Kravis, Peter Marks, Stuart Cobb

Marci Valner and Heidi Epstein, RSRT trustees

Bernhard Suter

Michelle Werner

Dina Katabi

Monica Coenraads

David Lieberman, Dina Katabi, Eric Marsh

Bob Deans, CTO, RSRT

Christy Zigler, Liz Berry-Kravis, Randy Carpenter, Paul Wang

Nasha Fitter

Cary Fu, Bernhard Suter, Jana von Hehn

Taysha team - Sukumar Nagendran, Emily McGinnis, Benit Maru, Rumana Haque-Ahmed, Sean McAuliffe

RSRT team - Tony Schoener, Stephanie Bohn, Marci Valner, Adrian Bird, Heidi Epstein, Monica Coenraads, Jana von Hehn, Bob Deans, Randy Carpenter

Kyle Fink and Shawn Liu

Eleanor Freeman and John Sinnamon

Eleanor Freeman, Adrian Bird, Harrison Gabel, Megan McGill

David Lockhart

Jana von Hehn, PJ Brooks. Danielle Brooks, Mustafa Sahin, Victor Faundez, Guoping Feng

Erik Sontheimer, Jonathan Watts, Scot Wolfe

Adrian Bird, Jana von Hehn, Bob Deans

Kyle FInk and Fyodor Urnov