Thousands of promising gene therapies for rare diseases stall before reaching patients due to lack of funding for first-in-human trials. Small patient populations deter traditional investors, leaving families to raise millions for trials while their children deteriorate from rapidly progressing, often terminal conditions. And yet, once validated with human data, these therapies can—and have—become billion-dollar products. Kylix Bio was founded to address this structural failure in rare disease. We identify high-potential gene therapy programs, fund them through first-in-human trials, and exit at a de-risked, value-inflected stage. We are currently advancing two programs toward clinical trials for neuromuscular diseases that currently have no treatment. We are ending the era where a child's life depends on a bake sale.