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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.


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.