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Details on Neurogene Gene Therapy Trial Now on

June 12, 2023

We've had two exciting gene therapy updates just in the last eight days. Last Monday, Taysha Gene Therapies announced that the first patient in their clinical trial has been dosed. Today, Neurogene shares new information on its gene therapy clinical trial, now on, including the sites where the trial will occur and a Rett community letter.

RSRT has worked very closely with Stuart Cobb, PhD, for over a decade and provided millions of dollars to his lab at the University of Edinburgh for the research that generated the gene therapy being used in the Neurogene trial. Dr. Cobb became Neurogene’s chief scientific officer following an introduction by RSRT to Neurogene’s CEO, Dr. Rachel McMinn. We are grateful to everyone at Neurogene for their passion and investment in the Rett syndrome gene therapy program.

This news is also the tangible results of the generosity of our donors and the actions of so many parents, grandparents, and families that have fundraised for RSRT. The dollars you contributed and raised played a critical role in making this happen. I remind our community that our work is far from done. I can’t emphasize that enough. Only when we have a genetic-based medicine that clearly shows dramatic improvement for all our loved ones will we have achieved our goal. That day is not here yet, but it will get here sooner with more families taking action.

RSRT’s track record in advancing genetic medicines is indisputable. Join our effort and be part of this exciting momentum.

Monica Coenraads

Neurogene Gene Therapy Clinical Trial Summary Protocol

Neurogene Rett Syndrome Community Letter