An independent AI lab for rare disease
One rare disease is rare. All rare diseases are not.
It started with my son's genetic hearing loss. Now it is a lab for the rare diseases the market skips.
Rare, one at a time. Common, all at once.
Any single rare disease is, by definition, small. Put them together and they are one of the largest health problems on earth. That is the whole thesis. The hard part is not any one disease. It is that the work does not compound across them. Registries do not talk. Tools get rebuilt. Each family starts from zero.
Sources (3)
- Nguengang Wakap S, et al. Estimating cumulative point prevalence of rare diseases. European Journal of Human Genetics, 2020.
- NIH Genetic and Rare Diseases Information Center (GARD), FAQ.
- The National Economic Burden of Rare Disease Study. EveryLife Foundation and Lewin Group, 2021 (US cost of rare disease: $966B in 2019).
How MISHA works
The bridge, and the guard on the bridge.
Three groups need each other and cannot connect. Families live the disease but cannot turn it into research. Researchers can test ideas but are scarce and flooded with noise. Patrons can fund it but need to see leverage. MISHA is the connective tissue between them, and the filter that keeps it honest.
Families
Bring the case and the urgency. We turn it into a real hypothesis, and recruit family ambassadors to lead new disease branches.
For familiesResearchers
Test what we bring. We guard your time: only computation-verified, wet-lab-ready hypotheses reach you, never unfiltered noise.
For researchersPatrons
Fund the bridge. Early capital compounds into the grants, the registry, and the first lab partnership.
For patronsHow we work
The real product is a research pipeline. Hearing loss is where we point it first.
We test one AI-augmented research pipeline on open problems in pure mathematics, where a proof kernel checks every step, and point the same pipeline at rare genetic disease. The method is the product. The disease is the proof.
How the pipeline worksWhat the foundation does
Four programs. Two build the science and its data, one moves money to the researchers who can use it, one holds the community together. Each is scoped so the tools outlast any single disease.
Research Lab
An AI and computational arm that synthesizes global work, ranks the hypotheses worth testing, and publishes finished outputs open-access.
Read morePatient Registry
Family-controlled, IRB-approved records and natural-history data, federated so many small cohorts become one research surface.
Read moreGrants Program
Seed grants to external researchers, sized to unlock the far larger grants that follow. This is venture philanthropy, not charity.
Read moreThis one started at a hearing test.
My son Misha was diagnosed at age two with bilateral sensorineural hearing loss caused by mutations in the STRC gene, the condition known as DFNB16. STRC is the second most common genetic cause of this kind of hearing loss. It is common enough to matter and rare enough for the drug industry to skip. That gap, between a solvable problem and a funded one, is where we work.
There is proof this is solvable. In 2026, a dual-AAV gene therapy restored hearing to near-normal thresholds in a DFNB16 mouse model, and the animals regained normal frequency discrimination for up to 100 days. The biology is tractable. What is missing is coordination and money, not possibility.
Read the mission