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Attacking Rett Syndrome From Every Angle

A cure for Rett Syndrome. You want it. We want it. More than anything. Together, we can make it happen.

From 2017 to 2020 we carried out Roadmap to a Cure, a research plan that identified and advanced every possible genetic strategy that targets 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 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:

The root cause is known
The root cause is known — alterations in a single gene, MECP2
The disorder is not neurodegenerative
The disorder is not neurodegenerative — brain cells don’t die
Restoring levels of the MECP2 protein dramatically reverses symptoms in mice
Restoring levels of the MECP2 protein dramatically reverses symptoms in mice

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.

  • Gene Replacement
    The 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 Editing
    Rather 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 Reactivation
    Reactivation could mitigate the flawed MECP2 gene by reawakening its silenced counterpart, which is present in all females.
  • RNA Editing
    RNA 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-splicing
    This strategy harnesses a naturally occurring process to replace the section of MECP2 RNA that contains most mutations with a healthy replacement.
  • Protein Replacement
    Rather than target the gene or the RNA, this strategy delivers the healthy MECP2 protein on an ongoing basis.

MECP2 Therapeutic Pipeline

RSRT’s support, prior, during, and after Roadmap to a Cure, has resulted in an extensive pipeline of MECP2 therapeutic programs now in development at biopharmaceutical companies and in academia. Every single company pursuing a cure for Rett is able to do so because they leveraged discoveries and resources that were incubated with RSRT funding.

This pipeline, which shows current and projected 2024 goals, is dynamic. As new data emerges the pipeline will be updated.

  • Research & Discovery
    The earliest stage of development, encompassing basic research to identify therapeutics.
  • Proof of Concept
    Preliminary experiments, typically performed in test tubes, cells or mice, that confirm the expected beneficial therapeutic effects.
  • Safety & Manufacturing
    Required studies that assess safety, tolerability, efficacy, and dose ranges prior to initiating human studies.
  • Clinical Trials
    Testing 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 Therapeutic
    Therapeutic 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 academic scientists are not drug developers — biopharmacuetical companies develop 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 biopharmaceutical companies below to initiate MECP2 therapeutic programs.

Accomplishments

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Biopharma

RSRT-sponsored research led to industry pursuit of multiple Rett programs.
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Therapeutic

RSRT funding introduced and expanded novel therapeutic areas for Rett that did not exist before.
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Million

Raised $33 million in three years to deliver on Roadmap to a Cure.