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Six Strategies, One Goal

CURE 360: An equal focus on science and business development

To cure Rett syndrome, RSRT recognizes that focusing on science alone is not enough. Careful thought and planning must also be paid to the business development side of our efforts.

How Drugs Are Developed

The spark for a new drug typically happens with a basic science discovery in an academic laboratory. Sometimes drugs are discovered by accident (e.g., penicillin), other times through a deliberate effort. A drug candidate is then tested in cell cultures and then in animal models. With encouraging data in hand, intellectual property (IP) is filed. Scientists sometimes start a company that licenses the IP from the academic institution. More often, however, the institution markets the IP to biopharmaceutical companies in search of a home for it. Once the IP is licensed, the biopharmaceutical company begins work on a disease indication.

Our goal is to ensure that this process of transitioning from research lab to biopharma occurs swiftly and efficiently. The divide between academia and biopharma is often referred to as the “Valley of Death” because most discoveries never make the transition. RSRT’s CURE 360 eliminates the “Valley of Death” by bridging this divide through our prodigious scientific and business connections, and drug development experience.


Professor Sir Adrian Bird’s landmark reversal experiments and subsequent MECP2 gene replacement studies resulted in symptom improvement much greater than that of any drug or compound tested to date. For this reason, and because Rett is a single-gene disorder with no evidence of brain cell death, RSRT is MECP2-centric in its approach to therapeutic strategies.

CURE 360 attacks the root cause of the disease from every angle. We have no doubt the cure will come from one of these six priority research strategies.

Six Strategies

Gene Replacement

Gene replacement introduces healthy copies of the MECP2 gene into the body to compensate for the mutated one and is the strategy that is closest to the clinic. RSRT-funded research through our Gene Therapy Consortium, launched in 2014, has resulted in Taysa Gene Therapies pursuing a gene therapy replacement programs for Rett syndrome as well as several companies who have not yet discolsed their programs. We are also advancing two academic replacement programs, one at the University of Pennsylvania led by pioneering gene therapist James Wilson and the other at the University of Edinburgy led by Stuart Cobb.

Gene Editing

Rather than introduce extra healthy copies of a gene, this strategy fixes the underlying mistake in the MECP2 gene using a technology that has taken the scientific world by storm: CRISPR. We support two efforts in this space: one at Beam Therapeutics, a leading DNA base editing company, and another at University of Massachusetts Medical School.

MECP2 Reactivation

MECP2 is on the X chromosome, and all females have two Xs. Beside each active, mutated gene rests a healthy but silent twin. Reactivation could mitigate the flawed gene by reawakening its silenced counterpart. RSRT has championed research in this area since our launch in 2008.

RNA Editing

RNA is a copy of DNA, and therefore any mutations found in a gene are also found in its RNA. We are advancing a number of RNA editing efforts, all of which harness normal editing processes that already exist in our cells.

RNA Trans-splicing

This strategy also harnesses a naturally occurring biological process to splice out the section of MECP2 RNA where almost all the mutations are and splice in a healthy replacement.

Protein Replacement

Rather than target the gene or the RNA, this strategy delivers the end product, the MECP2 protein. Rather than the “one and done” feature of the above strategies, protein will need to be delivered on an ongoing basis.

MECP2 Therapeutic Pipeline

RSRT’s support, prior to and during Roadmap to a Cure, has resulted in an extensive pipeline of therapeutic programs now in development at biopharmaceutical companies and in academia. 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.