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 small. Together they are one of the largest health problems on earth. The hard part is not any one disease. It is that the work does not compound: 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.
- Yang G, et al. The national economic burden of rare disease in the United States in 2019. Orphanet Journal of Rare Diseases, 2022 (total burden: $997B).
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, 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.
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.
One AI-augmented pipeline, tested where the answer is checkable: eleven machine-verified formalizations merged into the community Lean pool, fixes merged into RDKit and SageMath, and the same method pointed 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.
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. Venture philanthropy, not charity.
Read moreThis one started at a hearing test.
My son Misha was diagnosed at 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: common enough to matter, rare enough for the drug industry to skip. That gap is where we work. MISHA stands for Medicine, Intelligence, and Science for Human Action. The honest expansion is shorter: it is his name, and the foundation is scoped for the thousands of families who carry the same and adjacent variants.
There is proof this is solvable. In 2026, a dual-AAV gene therapy restored hearing to near-normal thresholds in a DFNB16 mouse model, with recovery durable to at least 100 days after injection. What is missing is coordination and money, not possibility.
Read the mission