Each rare disease affects relatively few people. Together, they affect millions — and for most, there is still no approved treatment. Rare disease is the heart of Koru's work.
In the UK, a disease is rare when it affects fewer than one in 2,000 people.
rare diseases have been identified.
people will be affected by a rare disease at some point in their lives.
Source: The UK Rare Diseases Framework, Department of Health and Social Care (2021).
Patients are few and spread across countries, which makes trials hard to run.
Natural history and biology are often poorly documented.
Many patients wait years for a correct diagnosis, and treatment waits with them.
The cost of a new molecule rarely fits a small market, so many conditions go unaddressed.
The initiative brings together our repurposing science, our stratification approach and our partners to deliver credible treatment options for rare conditions faster.
We look first at medicines with existing human data, to reach patients sooner.
Genetic and molecular markers guide which patients each programme is for.
Patient organisations help shape our priorities, endpoints and study design.
Where we can, we share findings with the research community, including what did not work.
We plan early for orphan designation and expedited regulatory routes in the UK, EU and US.
Understand the disease through patients, families, clinicians and the published evidence.
Identify the genes, pathways and markers that drive the condition and define patient subgroups.
Search existing compounds for those that act on that biology.
Test the strongest candidates in relevant models with academic partners.
Design small, precise studies and a regulatory route built for rare disease.
Help us understand what matters most to your community.
Share insight on unmet need and potential study sites.
Collaborate on models, biomarkers and validation.
Explore a new rare-disease indication for your compound.