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RAREplatform

RAREplatform is a patient-centric pathway that brings promising academic therapies to the patients who need them.

WinQure Medicine: RAREplatform

HOW IT WORKS

A five-step patient-centric pathway from discovery to patients

Academic therapies provide the scientific starting point. RAREkey helps us identify patients early and understand their needs. RAREscore assesses where therapies could make the greatest difference, while our in-house expert review examines the evidence and the path to clinical development.

WinQure then brings these elements together, working with researchers and patients to advance therapies from the laboratory toward treatment.

RAREplatform

Five connected steps to bring promising therapies from academic research to the patients they were developed to treat.

  1. Academic therapies

    Giving promising research a path forward

    We work with academic researchers who have developed promising ASO therapies but need support to take them beyond the laboratory and into clinical development. Researchers can share their existing data in any format, with no need for extensive preparation or special formatting.

    Contact WinQure to collaborate
    Step1
  2. Step2

    RAREkey

    Making patients visible from the beginning

    RAREkey is WinQure’s free patient registry, open to patients with a confirmed genetic diagnosis and their parents or legal guardians. Registration takes just a few minutes and makes patients visible for relevant treatment opportunities.

    Patients register their genetic variant, age, location and contact details. This allows WinQure to identify patients already waiting for treatment and take their needs into account when evaluating and initiating development programs.

    RAREkey also allows patients and families to share their needs and practical considerations, helping inform clinical trial planning before trials are designed.

    Submit a patient to RAREkey

  3. RAREscore

    Understanding where each therapy can make a difference

    RAREscore is WinQure’s proprietary AI-driven assessment tool, bringing together scientific evidence, development feasibility, expected clinical benefit and real patient data from RAREkey. It provides a systematic, evidence-based assessment of each therapy, identifying development opportunities, anticipating challenges and mapping the path from academic research to patients.

    Step3
  4. Step4

    Expert review

    Taking a closer look at each therapy

    Our team independently reviews the programs identified by RAREscore, examining the scientific evidence, development challenges and expected benefits for patients to determine the next steps toward clinical development.

    Read about the team

  5. WinQure program

    Bringing therapies to patients

    We in-license promising therapies from academic researchers and work with the originating researchers and clinicians, WinQure’s in-house scientific team and specialized contract organizations to advance them through pre-clinical development, manufacturing, clinical trials and regulatory approval, involving patients throughout the process.

    View our active programs
    Step5