Today’s experiment is
tomorrow’s potential cure
The Columbus Children’s Pipeline
Gene therapy is the only treatment with the potential to not only treat but actually cure genetic disease. Gene therapy involves giving patients an IV infusion filled with millions of copies of healthy genes that scientists have inserted into harmless virus molecules. These molecules target cells with the highest concentration of the faulty gene, allowing the healthy gene to take over from it. Once the healthy gene begins secreting the protein that the patient previously lacked, the disease process could slow considerably or even stop.
Working with our scientific advisory board and international partners, we are facilitating potential gene therapies into clinical research programs for ultra-rare neurodegenerative genetic disorders.
Ultra-rare diseases in the Columbus Children’s pipeline
CCF Open-Source™ expands treatment research
A key CCF initiative puts our program research and clinical data into the public domain where possible, building a shared knowledge base accessible to the gene therapy research community.
- Accelerates innovation with open access to current research
- Empowers the scientific community to advance ultra-rare gene therapy treatments
- Ensures data availability for any ultra-rare disease program
- CCF Open-Source is driving standardization into the clinical and regulatory processes