Attacking Rett Syndrome From Every Angle
From 2017 to 2020 we carried out Roadmap to a Cure, a research plan that identified and advanced six therapeutic strategies targeting the root cause of Rett Syndrome.
CURE 360 is the blueprint for how we now drive those advances into a coordinated, full-on attack on Rett.
We know scientific discoveries are made in academic labs and drugs are developed by biopharma. So CURE 360 is focused intensely on research and business development. CURE 360 further evolves the Rett Syndrome Research Trust (RSRT) from a disease research organization into an MECP2 therapeutic incubator, ensuring that biopharma picks up academic programs advanced during Roadmap to a Cure.
The cure will come from one or more of the therapeutic strategies encompassed in CURE 360 and described below. We invite you to be part of the solution. Contact us today.
Learn how RSRT is accelerating the attack on Rett Syndrome with Cure 360.
Rett is surrounded.
We’re closing in.
Despite its debilitating nature, Rett offers three silver linings:
Together, these three facts scream out for a genetic solution to Rett. CURE 360 provides six potential solutions unified by a common objective: restoring levels of MECP2 protein.
There is a lot going on now in the world of Rett research and the best of it is supported by RSRT.
MECP2 Therapeutic Pipeline
RSRT’s support, prior to and during Roadmap to a Cure, has resulted in an extensive pipeline of MECP2 therapeutic programs, now in development in biopharma and academia.
This pipeline, which shows current and projected 2024 goals, is dynamic. As new data emerges the pipeline will be updated.
RSRT’s Role As An MECP2 Therapeutic Incubator
Breakthrough discoveries are made in academic labs. But academics are not drug developers — biopharma develops drugs. As Rett Syndrome is not low-hanging fruit for biopharma, RSRT functions as an MECP2 therapeutic incubator, by ensuring that biopharma picks up programs that we have advanced. The basic science, translational efforts, tools and resources we have nurtured and supported have facilitated the biopharma companies below to initiate MECP2 therapeutic programs.
Accomplishments
Our Research Collaborators
By compiling an extensive repository of research tools and ensuring these resources are widely available to scientists, RSRT is accelerating basic and translational research efforts in a meaningful and tangible way.
For Researchers
Connecting academic and industry scientists with the resources and reagents needed to make quick progress is important to us. Please visit our curated list of key resources and reagents.

A cure for Rett.
You want it.
We want it.
Great achievements happen when people stand up and say I’m in. I will be part of the solution!
Rett may be a rare disorder but together we are powerful.