Why we exist.
The HIKESHI Foundation was founded by families who refuse to accept that their children must die from a disease that science can cure. We exist to bridge the gap between groundbreaking research and lifesaving treatment.
What we do.
Three pillars of work that move us toward a cure.
Fund the science
Our goal is to channel grants to gene therapy research that can translate proof-of-concept into a real treatment.
Build the registry
Connect every affected family worldwide so no child is invisible to research.
Expand screening
Add HIKESHI to standard Ashkenazi genetic screening so carriers know their risk.
Our 6-year roadmap.
From foundation to FDA approval — a defined, fundable path.
- 1Year 1
Establish foundation, build patient registry, raise initial $2M.
- 2Year 2
Fund preclinical animal studies, secure FDA Orphan Drug Designation.
- 3Year 3
Partner with biotech for GMP vector production, prepare IND application.
- 4Years 4–5
Phase I/II clinical trials in affected children.
- 5Year 6
FDA approval and broad treatment availability.