Since the discovery and development of new, innovative genetic medicines focus mainly on larger patient populations, we strive to provide children with ultra-rare genetic conditions with the same level of access. CCF brings a network of scientific experts, technology resources, and affordable manufacturing capacity to expand options with three core areas of focus:

• Providing financial support to accelerate the advancement of gene therapy clinical trials for ultra-rare genetic diseases.
• Supporting the open-source data model and standard protocols across the broader gene therapy community.
• Removing the burden for children and families to access to life-altering clinical trials around the world.

CCF is building a bridge for children with ultra-rare disorders to ensure no child is left behind. The US-based Columbus Children’s Foundation was started in 2018 and operates in Chapel Hill, NC with collaborations throughout the U.S. and abroad.