OUR RESEARCH STRATEGY

Research Strategy & Investment

At the Cure MKD, we are committed to moving the field of Mevalonate Kinase Deficiency (MKD) beyond symptom management toward definitive, disease-modifying solutions.

Strategic Roadmap

Our mission is to accelerate the development of curative therapies by investing in high-impact research at accredited academic and biotechnology institutions worldwide. We prioritize projects that address the root cause of the disease and build the necessary infrastructure for successful clinical trials. Our funding strategy is organized into three primary pillars

Pillar 1 : Curative Solutions

Our highest priority is the development of therapies that provide a permanent solution to the genetic failure in MKD. We seek to fund innovation in the following areas:

Genetic Interventions: Advancing gene therapy and gene editing technologies designed to fix the MVK genetic failure at its source.

Enzyme & Pathway Restoration: Developing methods to replace the missing enzyme or restore the production of essential molecules (isoprenoids) that the immune system needs to stay regulated and prevent systemic inflammation.

Beyond Symptoms: Prioritizing any research aimed at a “solution-level” fix that addresses the underlying biological block rather than merely suppressing symptoms.

Pillar 2: Natural History & Clinical Readiness

To deliver a cure, we must have a standardized map of how the disease progresses. We invest in research that prepares the MKD community for the clinical trial stage:

Longitudinal Studies: Tracking the course of the disease over time to identify reliable biomarkers that regulatory bodies (such as the FDA and EMA) require for drug approval.

Clinical Endpoints: Determining exactly how to measure the success of a new treatment in a clinical setting, ensuring that once a cure is found, it can be approved and delivered to patients as fast as possible.

Pillar 3: Global Patient Registry

The success of future therapies depends on a robust and identified patient population. We support initiatives that expand our reach and find those who are still seeking answers:

Finding the Diagnosis: Supporting global efforts to identify MKD patients earlier through improved awareness and genetic testing access.

Building the Registry: Unifying patient data globally to ensure a reachable community when clinical trials begin.

SUBMIT A PROPoSAL

A Global Call for Collaboration

If you are a researcher or clinician with a project that aligns with our strategic goals, please download the template below and submit your proposal to isabella@curemkd.org.

Download template