About us
WinQure Medicine is a biotechnology company focused on developing antisense oligonucleotide (ASO) therapies for ultra-rare genetic diseases. We bring together scientific expertise, academic research and drug development experience to advance promising therapies that might otherwise never reach patients.
Promising therapies stall in academia
Advances in antisense oligonucleotide (ASO) technology have made it possible to develop therapies targeting specific genetic variants.
For many ultra-rare genetic diseases, therapy development begins in academic laboratories, where researchers develop promising ASO therapies, often with support from patient families and philanthropic organisations. Yet many of these programs never reach patients because academic institutions lack the development infrastructure needed to advance therapies through manufacturing, clinical trials and regulatory approval. As a result, promising therapies remain in academia despite their potential to help patients.
That is why WinQure Medicine was created.
Patients struggle to find relevant treatments
For patients with ultra-rare genetic diseases, finding and accessing newly developed or experimental therapies can be extremely difficult, particularly when clinical trials or treatment opportunities are available only in other countries.
At the same time, researchers and therapy developers often struggle to identify enough patients with a specific ultra-rare genetic disease to conduct clinical trials. As a result, patients may never hear about relevant treatment opportunities, while promising therapies may be delayed because eligible patients cannot be found.
That is why RAREkey was created.
Built to develop therapies for ultra-rare diseases
Developing therapies for ultra-rare genetic diseases requires specialized scientific expertise, close coordination and an approach suited to small patient populations. WinQure brings together experienced scientists, academic researchers, clinicians and development partners to advance promising therapies.
ASO expertise
Our scientific team brings decades of experience in antisense technology and drug development.
Academic network
Our global academic network provides access to expertise and promising research across genetic diseases.
Clinical partnerships
We work with specialist clinicians and hospitals to plan clinical development and identify treatment sites.
Regulatory expertise
Our team develops regulatory strategies suited to ultra-rare diseases and small patient populations.
Manufacturing capabilities
We work with specialized manufacturing partners to support ASO production, quality control and clinical supply.
Coordinated development
We coordinate expertise across development stages, ensuring continuity from academic research to clinical trials.
Our approach: From academic research to patients
WinQure Medicine develops antisense oligonucleotide (ASO) therapies for ultra-rare genetic diseases, building on promising research from academic laboratories around the world.
Through our RAREplatform, we bring together three essential elements: promising academic therapies, patients who may benefit from them, and the scientific expertise needed to advance their development.
Our free patient registry, RAREkey, helps identify patients with relevant genetic variants early in development. RAREscore, our proprietary AI-driven assessment tool, evaluates the scientific evidence, development feasibility and potential patient benefit of each program. Together with independent review by our scientific team, these tools inform which therapies we take forward, how we develop them, and when and where clinical development can take place.
We in-license selected academic programs and work with the originating researchers and clinicians, our in-house scientific team and specialized development partners to advance therapies through pre-clinical development, manufacturing, clinical trials and regulatory approval.
By building on existing academic research and sharing development expertise across programs, we aim to make the development of therapies for ultra-rare diseases more efficient and sustainable, bringing treatments to patients who have few or no options today.
Promising therapies already exist in academic laboratories. Our role is to bring together the science, patients and development expertise needed to take them forward.
