2025 Rett Genetic Medicines Summit: Key Takeaways
A recap of the science, collaboration, and lived experience that defined the 2025 Rett Genetic Medicines Summit—highlighting major updates, parent reflections, and new content now available to explore.
This September, RSRT brought together 150 leaders across biopharma and academia for the 2025 Rett Syndrome Genetic Medicines Summit. Over three days, the field aligned around one shared goal: advancing safe, effective genetic medicines that target the root cause of Rett syndrome.
The Summit created a focused environment for open, practical dialogue—where drug developers assessed the current landscape, scientists shared emerging insights, clinicians outlined real-world needs, and regulators clarified the path for advancing Rett programs toward the clinic. It underscored RSRT’s role as both curator and catalyst, helping translate promising ideas into meaningful progress.
Program Highlights
Speakers delivered clear, actionable updates across the genetic medicines landscape, offering a comprehensive view of the opportunities and challenges shaping Rett therapies. Poster sessions showcased new data from emerging scientists and industry teams, reflecting the breadth of innovation underway. Expert panels brought clinicians, regulators, and developers together to refine expectations, align on best practices, and discuss what it will take to advance genetic medicines responsibly and efficiently.
Most importantly 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.
Guided by Families, Grounded in Urgency
Parent reflections throughout the program kept individuals with Rett at the forefront. Their voices reminded attendees that this effort is fueled not by abstract optimism, but by hope grounded in science, urgency, and the push for transformative treatments.
What This Summit Makes Possible
The relationships strengthened at the Summit accelerate every program underway. They help prioritize the right science, advance IND-enabling work, and ensure the field stays aligned around what matters most.
As Monica shared in her opening remarks:
“Our children’s futures are in your collective hands.”
RSRT’s commitment is to make sure those hands have the data, clarity, and collaboration needed to turn promising ideas into real therapies.
Featured Videos
Summit Trailer
A look at the momentum behind genetic medicines for Rett.
Profluent x RSRT: AI-Designed MECP2
A breakthrough conversation on next-generation, AI-driven therapeutics, sparked at the summit.
Explore the Summit Hub
The Summit Hub features speaker highlights, company updates, scientific sessions, parent reflections, and additional program materials—and will continue to expand as more assets are released.
