We have exciting news to share. Taysha Gene Therapies has dosed its first Rett patient with gene therapy. This is an important moment for the entire Rett community. Not only is this Taysha’s first dosed Rett patient, it’s also the first time in history that a Rett patient has been given gene therapy. It’s a major step forward on the path to advancing genetic-based medicines that aim to profoundly change the lives of our loved ones.
A tremendous amount of energy, time, resources, dedication, perseverance, and grit has gotten us to this point. RSRT has championed gene therapy for Rett syndrome for nearly 15 years, with more than $10 million invested in this approach, including major funding to Steve Gray, PhD, who created a mechanism to control the level of MECP2 expression that is part of the Taysha product, and to our trustee and close advisor, Adrian Bird, PhD, who developed the MECP2 “mini-gene” that the Taysha clinical trial delivers.
When I talk about grit, perseverance and dedication, I’m including the families that fundraise for us. Without them, without their actions, I would not be reporting this news to you. These families made it possible to generate the foundational scientific data subsequently licensed by Taysha, which is now taking this program forward. To say I am profoundly grateful to these families and all of our loyal donors would be an enormous understatement.
This clinical trial is a notable step forward on our road to a cure. Key to achieving the cure we all envision for our loved ones will depend, in large part, on the future fundraising actions of the Rett community. We welcome the engagement of families who have not yet joined our efforts.
Please join me in thanking the brave family who agreed to be patient #1. We are sending this young woman our love and our best wishes.