

Rett Genetic Medicines Summit 2025
Over the course of three days in September we convened leading scientists, clinicians, and regulators to explore every corner of the genetic medicines landscape. The leadership from over 45 biopharma companies had a unique opportunity to hear about the state of Rett genetic medicine programs, with the ultimate goal of inspiring and motivating them to add the disorder to their pipelines.
This year’s gathering underscored RSRT’s role as both convener and catalyst, bringing brilliant minds together while never losing sight of the children and families at the heart of our community.

Summit Speakers

Platinum Sponsors



“I just wanted to say how much I enjoyed the meeting. I will admit I was tired by the end but there wasn’t a talk I could skip to have a rest, the programme was so good. The fact that I was wanting to get back to the lab to get stuff done tells you all you need to know.”


Summit Program PDF
Explore the full scope of Summit 2025 with our downloadable program, featuring session details, speaker bios, and an overview of the science presented.

Gold & Silver Sponsors
A Parent's Voice
Parents and trustees shared powerful reflections that grounded the science in lived experience. Their gratitude, urgency, and hope reminded us why this work cannot wait.

Bronze Sponsors
Alexion Pharmaceuticals • Alltrna • Apertura Gene Therapy
Ascidian Therapeutics • Biogen • Rgenta Therapeutics
uniQure • Wave Life Sciences
Event Photos


“I can’t thank you enough for all the effort you put into organizing such a fantastic summit. You brought together leading scientists across every area relevant to genetic medicines for Rett, creating an environment to share progress, resources, and explore collaborations. I learned so much from the meeting, and hearing directly from parents made me even more determined to continue on this scientific path to contribute to human health.”























































































