Making Gene Therapies Accessible to Patient with Ultra-Rare Conditions

While gene therapies provide great promise for people with rare, genetic diseases, the pursuit of these one-time treatments may not seem economically viable to commercial developers. In the case of ultra-rare conditions, the patient populations may be so small that it may not be possible for companies to recoup their investments, let alone make a profit. The nonprofit Columbus Children’s Foundation is providing funding and scientific resources to accelerate access to gene therapies for children with ultra-rare genetic disorders. We spoke to Jude Samulski, a gene therapy innovator and chairman and chief scientific officer of the Columbus Children’s Foundation, about the foundation operates, the range of capabilities it is able to bring together, and the potential to scale what it does to reach more patients in need of treatments.