The mission
One rare disease is rare. All rare diseases are not.
MISHA Foundation accelerates research into rare genetic disease by funding the labs that move fastest, building family-controlled patient registries, and putting computation to work where it moves biology forward. It started with one child's hearing loss. It is built to outlast his specific cure.
There are approximately seven thousand rare diseases and 350 million people afflicted by them worldwide. Approximately 95 percent of rare diseases do not have a single FDA-approved therapy because they are so poorly understood. Simply put, you can't create a targeted treatment when you don't know what needs to be targeted.
Why this exists
When you are told your two-year-old will not hear the way other children hear, you are also told, gently, to wait. Wait for research that no one is funding on your timeline. Wait for a trial that does not exist. Wait in a language no one has translated for parents.
STRC sits in a blind spot. Common enough to matter, rare enough to be ignored by commercial pipelines. The science is tractable. The structures are being modeled. The missing piece is not the biology. It is coordination, money moving to the right benches, and families who can find each other.
MISHA is named for one child and scoped for the thousands who carry the same and adjacent variants. I do not own the science. I fund it, connect it, run the computation, and keep the receipts in public.
Egor Lyfar, founder and Misha's father
How a small foundation moves a whole field
None of this is charity theatre. It is a specific, borrowed playbook. Four moves, in order.
Start where the gap is
Hearing-genetics research clusters in the West. We put the work in Asia, where Mainland trial pipelines meet Western lab quality, and where our family already lives.
De-risk with computation
Rank the hypotheses, model the structures, find the druggable pocket in silico first. The first wet-lab experiment should be the cheapest one, not the first guess.
Seed the labs that move
A 50k to 150k grant buys the preliminary data a lab needs to unlock a grant ten to fifty times larger. Repeated, that leverage is how the SMA and cystic fibrosis foundations reached their first therapies.
Keep the receipts in public
Every decision, every failed application, every ranking delta goes on the public wiki. For a one-person foundation, legibility is not a nicety. It is how you earn the right to hold real money.
Not an advocacy-only group, and not a single-target biotech. Closer in spirit to the early SMA Foundation, the Rett Syndrome Research Trust, and the Usher 1F Collaborative: small, technical, and pointed at tools the whole field can use.
Where computation actually helps
This is not AI as a slogan. It is a specific bet about which parts of rare-disease research got cheap, and using that to spend wet-lab money last, not first.
Structure used to take years
Predicting a protein's shape once meant months or years at a crystallography bench. AlphaFold has released more than 214 million predicted structures, and its authors shared the 2024 Nobel Prize in Chemistry. Inference now runs in hours.
Nature 2024; Nobel Prize 2024
Rare disease is mostly monogenic
About 72% of rare diseases are genetic, usually a single gene. That is the tractable case: model the variant, rank the hypotheses, find the druggable pocket, before anyone pipettes anything.
Nguengang Wakap et al., 2020
Small cohorts add up when federated
One rare-disease registry is too small to power a study. Many registries, sharing a common structure, become a research surface big enough to matter. The FDA now has guidance on using natural-history data as an external control arm.
FDA Natural History Studies guidance, 2019
A bridge, and a guard on the bridge
Families carry the case. Labs can test it. Between them sits a gap that wastes both. We close it, and we guard it: a family's case becomes a rigorous, computation-verified hypothesis, and only the serious ones reach a researcher's bench, never unfiltered noise. That is the whole job.