Meeting a significant unmet medical need
Every child deserves to live life to its fullest
Most pharmaceutical and life science companies focus on genetic disorders with greater commercial potential thus, ultra-orphan diseases are often overlooked because the economics don’t add up. Our aim is to bring funding and scientific resources to the forefront so ultra-rare genetic disease research and development progresses at the same pace as other larger indications. Children with these diseases and their families urgently need access to transformative genetic medicine. Most are suffering a severely altered way of life and, in many cases, life expectancy is shorter than ten years.
3 of 10 children with a rare disease won’t live to see their 5th birthday
“If there is a promising path to develop a curative gene therapy solution, we must lead the way to bring treatments to the clinic for children who desperately need them. We are deeply driven by the urgent need to make sure no child is left behind.”
R. Jude Samulski, Ph.D., Chairman and Chief Scientific Officer
Overcoming scientific and financial barriers for ultra-rare diseases
When development costs are spread over a very small population of patients, it limits the ability for profit-driven life science enterprises to bring ultra-rare therapies to market. Thus, progress is too slow and the cost is too great. We must accelerate research for ultra-rare diseases and mitigate the challenges for children with the highest need to participate in clinical trials.
Less than 20 in 1,000,000 have an ultra-rare disease
All children should have access to needed gene therapy medicine.
- We collaborate with scientific and clinical experts to advance ultra-rare programs into clinic
- We partner internationally with gene therapy manufacturers lower the cost of production and secure capacity
- Our non-profit model removes commercial ROI expectations that comes with gene therapy technology and clinical processes
Through the generosity of philanthropic and patient advocacy organizations combined with the the scientific backing of the genetic science and medical community, we are bringing focus to ultra-rare conditions while mitigating the cost associated with therapeutic development. Every child fighting the devastating effects of genetic diseases deserves our attention when a cure is in reach.
“Money shouldn’t stand between children with ultra-rare diseases and treatments that could possibly cure them. The Columbus Foundation is bringing hope to deserving kids and their families who need all that gene therapy can offer them.”
Sheila Mikhail, Founding Trustee, Columbus Children’s Foundation
Ultra-rare neurodegenerative genetic diseases are hereditary and arise from faulty or missing genes passed on from a parent. They severely affect movement, speech, memory and other vital functions with rapidly increasing disability. Current treatments are not curative and only address symptoms thus, life expectancy for many with ultra-rare diseases is rarely beyond early childhood.
There is an urgent need for ultra-rare disease research and therapies to change the lives of children around world.
Marta M., Spain
Marta M. was just two months old when she was diagnosed as only the 100th child in Spain with Niemann Pick C disorder, an incurable neurodegenerative hereditary disease with a life expectancy of at best 20 years. Thanks to the support of the Fundacion Columbus, our European counterpart, Marta now has a life filled with hope.