Deadline: 01-Oct-2025
The EQT Foundation has launched a new call for proposals under its Breakthrough Science Program to award researchers tackling urgent challenges in the field of rare diseases.
Designed to accelerate bold scientific ideas with high potential for real-world patient impact, the program supports translational research that might be overlooked by traditional funders. The EQT Foundation awards between EUR 25,000 to EUR 100,000 to support groundbreaking research in the field of rare diseases.
The program is 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 they 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.
Geographical: Any scientist/researcher associated with an accredited nonprofit University, hospital, or research institution around the world is eligible to apply. Science and Technology: The concept is based on novel scientific or deeptech technology. The scientific rationale and documentation validate the idea, and the intended scientific outcomes solves a huge challenge with potential for creating high impact while addressing specific rare diseases. They’re placing an emphasis on funding high-risk, high-reward proposals that might not otherwise get funded. Entrepreneurial Spirit: The proposed solution addresses a significant or future market need; the applicant can articulate the insights and assumptions they’re basing their vision on; and demonstrates the courage, creativity, and drive to pioneer new approaches and translate discovery to impact. Ideal candidates will have: Strong academic track record and bold science or deeptech innovation potential to make a significant difference towards diagnosing and treating of rare diseases; An entrepreneurial mindset with a demonstrated or clearly articulated vision for translating their research into the market for patient impact. More than 300 million people worldwide live with a rare disease, nearly half of them children. For many, the journey to diagnosis is prolonged and uncertain, with limited or no treatment options available. Despite these challenges, the rare disease sector has contributed to transformative breakthroughs in medicine, from gene therapies to RNA-based treatments.
The foundation is not only funding research but also empowering bold scientists to move their work from lab to clinic. The aim is to foster innovations that improve treatments for rare diseases in cost-effective ways, presenting opportunities for impact investors to enhance quality-adjusted life years.
Beyond funding, selected grantees will gain access to EQT’s extensive global network, offering connections to advisors, industry partners, and commercialization support specifically tailored to translational science.
Applications for the program open on September 2, 2025, and close on October 1, 2025, at 23:59 CET. A panel of scientific and translational experts will review proposals, with shortlisted candidates invited for interviews with the EQT Foundation team. Final decisions will be announced shortly thereafter.
For more information, visit EQT Foundation.
