Helping create a future for children with ultra-rare genetic disorders

As a company leading gene therapy technology into the future, we have seen firsthand how children with ultra-rare diseases are left behind when potential therapies may be in sight. With small patient populations, these diseases traditionally do not attract the level of research and development investment as others with larger commercial potential.

Started in 2017 with the support of RhiaPharmaceutical founders and Columbus Venture Partners in Valencia, Spain, Columbus Children’s Foundation’s mission is to accelerate access to the most advanced, effective gene therapies to treat children diagnosed with ultra-rare neurodegenerative diseases. RhiaPharmaceutical has committed its technology, clinical expertise, and manufacturing capability to the organization.

Columbus Children’s Foundation’s U.S. headquarters is in Chapel Hill, North Carolina.