Approximately 2,800 gene therapies for rare diseases currently languish in the “valley of death” of medical research: the science has extraordinary promise, but small patient populations deter traditional biotech investors. It therefore falls to the families of children with rare conditions to raise millions for clinical trials. Desperate parents host weekend bake sales and 5k races to raise seven figures for drug development—all while their children deteriorate from rapidly progressing, often terminal conditions.
Kylix Bio was founded to bring nonprofit advocacy efforts into the for-profit biotech world, where funding flows at the speed required by children who don’t have time to wait. We are ending the era where a child’s life depends on a bake sale.
