Stuart Cobb (above) will be a familiar name to many of you. His introduction to Rett syndrome came courtesy of Adrian Bird as co-author on the now famous reversal experiment paper, which demonstrated that Rett symptoms were reversible in mice. With almost $3 million in RSRT funding, Stuart has explored a variety of strategies to boost levels of MECP2 protein in the brain, including gene replacement, RNA trans-splicing, and protein replacement.
He was one of the four members of RSRT’s original Gene Therapy Consortium, along with Steve Gray, PhD, Brian Kaspar, PhD, and Gail Mandel, PhD. Remarkably, all of the academic Rett programs pursued by these four scientists have now transitioned to companies. Steve Gray’s program was licensed by Taysha Gene Therapies, Brian Kaspar’s by AveXis (later terminated by Novartis), Stuart Cobb’s by Neurogene, and Gail Mandel’s RNA editing work by Vico Therapeutics.
Today’s announcement is a prime example of the impact of the families that fundraise for RSRT and your donations. You made it possible for RSRT to commit $7 million to these four investigators. Those dollars are now being leveraged by these companies to develop clinical trials and, we hope in the not-too-distant future, change our children’s lives. This is also an opportunity for me to thank the RSRT board for its confidence and trust in our team, as well as international Rett organizations such as Reverse Rett in the UK, the Rett Syndrome Research & Treatment Association in Israel, and Rett Syndrome Deutschland, which have been especially supportive of our gene replacement efforts. We wouldn’t be where we are without each of these very important constituents.
Developing a genetic-based medicine is difficult and risky. I am thrilled that we now have an eighth company pushing aggressively to advance their Rett program. The richer the pipeline of development programs, the better off our community is.
Please join me in welcoming Neurogene to our community and wishing them Godspeed.
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