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.
CURE 360 further evolves RSRT from a disease research organization into an MECP2 therapeutic incubator.
Rett is surrounded.
We’re closing in.
Despite its debilitating nature, Rett offers three silver linings:
- Gene ReplacementThe curative strategy closest to clinical trial, gene replacement introduces healthy copies of the MECP2 gene into the body to compensate for the mutated one.
- Gene EditingRather than introduce extra healthy copies of a gene, this strategy fixes the underlying mistake in the MECP2 gene using state-of-the-art CRISPR technology.
- MECP2 ReactivationReactivation could mitigate the flawed MECP2 gene by reawakening its silenced counterpart, which is present in all females.
- RNA EditingRNA is a copy of DNA, so mutations in a gene are also in its RNA. RNA editing efforts harness normal editing processes that already exist in our cells.
- RNA Trans-splicingThis strategy harnesses a naturally occurring process to replace the section of MECP2 RNA that contains most mutations with a healthy replacement.
- Protein ReplacementRather than target the gene or the RNA, this strategy delivers the healthy MECP2 protein on an ongoing basis.
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.
- Research & DiscoveryThe earliest stage of development encompassing basic research to identify therapeutics.
- Proof of ConceptPreliminary experiments, typically performed in test tubes, cells or mice, that confirm the expected beneficial therapeutic effects.
- Safety & ManufacturingRequired studies that assess safety, tolerability, efficacy and dose ranges prior to initiating human studies.
- Clinical TrialsTesting of the therapeutic in humans, first for safety and then for efficacy. This may be done in a series of trials with a single objective, or in single trials with multiple objectives.
- Available TherapeuticTherapeutic has demonstrated safety and efficacy and can be commercialized by companies and used by doctors to treat patients outside of a clinical trial.
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.
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.
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.