The Rath Family Foundation is committing $3 million over the next three years to research on brain and spine hemangioblastoma, with $3–10 million of venture capital behind any result that starts to look like a therapy. A conventional grant program posts a call and waits. In a compartment with no immortalized cell line and no animal model of the CNS lesion, waiting produces relabeled kidney proposals. So the project works from a published agenda: it finds who already holds the cohort, the tissue or the method each direction needs, and commissions the experiment — and it funds inbound proposals that genuinely move the same agenda.
For directions where the material already exists and nobody is asking the question — a stain on archived blocks, a reanalysis of serial imaging, a copy-number assay on banked DNA, a data linkage across two registries — the project specifies the experiment, pre-registers the readout, and pays the full direct cost to the group best placed to run it.
Most of the agenda's highest-value items are in this category, and most cost under $75,000 and need no new patients.
Where a group has built a cohort, a pipeline or a platform for another VHL compartment — a single-cell atlas of renal tissue, a response-prediction model in pancreatic NETs, a retinal AAV model — the project pays the marginal cost of pointing it at hemangioblastoma. The lab keeps its program, its IP and its publication; the project buys the one additional question.
Any group can propose work that addresses a direction on the agenda, or makes a case for one that should be on it. One-page proposal, rolling, a decision with a reason inside 60 days. The bar is the same as for commissioned work: a plausible path to fewer lifetime surgical procedures for people with VHL, and evidence that the approach reaches a hemangioblastoma of the brain or spine.
These are the rungs as they exist across rare disease, from a researcher with an idea to an approved therapy. The project pays for the middle two. Select a rung to see what sits on it.
The honest version: a $3 million grant tier reaches readiness assets and repurposing trials. The $5–12 million IND-enabling step needs the venture tier or pooled co-funders. Pooling by shared bottleneck rather than by disease is how thousands of $200,000 foundations become one $12 million package.
Those are the rungs as the field has them. The project’s own tiers start one rung lower: it pays for the cheap, decisive tests that have to happen before there is a discovery worth funding, and it keeps the grant tier non-dilutive so nothing about the money distorts the science.
The grant tier is funded by the Rath Family Foundation. Every award, result and cost is published on this site so the next condition’s pool starts with a map. Other funders, labs and families can join at the same rung; the structure is built to be copied.
Use the contact form, and tell us which direction on the agenda your group could answer.