Open call for applications: Rare Diseases

About

Over 300 million people worldwide live with a rare disease - nearly half of them children. For most, the journey is marked by years-long diagnostic odysseys, frequent misdiagnosis, and a lifetime of symptoms with no approved or effective treatments. The result is profound suffering and an urgent unmet need.

EQT Foundation is seeking to award €25,000 to €100,000 in catalytic grant funding to research projects with innovative, translational approaches that have the potential for outsized patient outcomes in this space. Time and again research and solutions developed for the rarest conditions - gene editing, targeted biologics, next-generation diagnostics - have gone on to transform the lives of those impacted by rare conditions whilst revolutionizing medicine and the treatment for far more common conditions. We’re looking for scientists and researchers with entrepreneurial potential who are developing solutions that can move from lab to market - addressing specific and urgent unmet needs within rare diseases, while advancing the frontiers of science and medicine globally.

Selected projects will receive not only funding, but support from the EQT Foundation including access to EQT’s global network of healthcare experts, potential industry partners, and founder/commercialization support.

We are particularly interested in proposals focused on:

• Novel therapeutic platforms & curative modalities:
  • Bold approaches that treat rare diseases at their root cause with a clear path to IND/first-in-patient. This includes enzyme replacement therapies, oral therapies (e.g. neurotransmitter modulators), gene therapies, RNA-based therapeutics (e.g. RNA-based gene modulation, RNA interference), synergistic approaches, and delivery engineering (e.g. alternative delivery approaches, vector optimization, CMC, in-process analytics), and n-of-few/one toolchains that responsibly scale individualized medicines.
• Biomarkers for trial enablement, with selective early detection:
  • The discovery, validation, and development of assays for novel prognostic biomarkers and candidate surrogate outcome measures that improve small-population trial design. Instrument-light or ultra-rapid molecular assays using new chemistries or microfluidics that materially shorten the diagnostic odyssey for patients in underserved settings and have a clear path to impact.
• Trial & evidence acceleration (path to first-in-patient):
  • Deeptech methods that turn scarce, fragmented and small-N data into regulatory-grade evidence and accelerate the path to first-in-patient within 12-18 months. Examples could include FAIR-aligned, interoperable registries for a specific rare disease network; submission ready natural-history and digital biomarker platforms; AI-enabled drug repurposing with a clear clinical evidence plan for new indication.
• Access-enabling technology:
  • Rare diseases affect people worldwide, and we encourage ideas that benefit patients in low-resource or remote settings. This could mean developing cost-effective therapies or diagnostics that can be deployed in healthcare systems with limited infrastructure, or process innovations that lower cost and complexity of manufacturing/distribution suitable for constrained settings and tied to measurable patient outcomes for individuals with rare conditions.
• Innovative disease models & mechanistic insights:
  • Human-relevant models (e.g., patient-derived iPSCs, 3D organoids, organ-on-chip) that recapitulate rare-disease biology and de-risk targets/therapeutics—enabling biomarker discovery, patient stratification, and decision-grade preclinical evidence.

Out-of-scope proposals include, but not limited to proposals that:

• Involve OTC/supplemental products
• Repurpose already marketed therapies without a robust mechanistic and clinical evidence plan for the new indication
• Focus solely on telemedicine, operational efficiency, or wellness applications without direct, measurable impact on rare disease diagnosis or patient outcomes
• Are purely observational, social science & community programs, or basic research without a credible translational pathway