Cure Rare Disease Announces Partnership with Columbus Children’s Foundation

Companies Unite in an Effort to Drive the Development of Drugs for Rare Diseases Globally

Boston, Massachusetts–(Newsfile Corp. – August 19, 2020) – Cure Rare Disease (CRD), the emerging, Boston-based nonprofit on a mission to develop customized therapeutics for rare diseases, announced a strategic partnership with Columbus Children’s Foundation (CCF), a nonprofit biotech known for its role in the advancement and acceleration of gene therapy programs bringing hope to children with ultra-rare genetic diseases. In addition to advancing its internal programs for ultra-rare diseases, CCF is partnering with contract development and manufacturing organizations (CDMOs) such as Viralgen Vector Core (VVC), a leading manufacturer of Adeno-associated virus (AAV) vectors for gene therapy, to provide, expertise, access to capacity and significantly discounted Adeno-associated viruses (AAV) for ultra-rare diseases and mutations for which there’s limited therapeutic availability.

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Key Takeaways

  • Cure Rare Disease is partnering with Columbus Children’s Foundation to address the economic accessibility issues that nonprofit ultra-rare drug development programs face.
  • Manufacturing is one of the most difficult steps and CCF is helping to close the rare disease divide.
  • Working with CCF and their partners, like VVC, offers access to support in the AAV field, paired with a high-quality product that’s being produced for the first custom treatment for Duchenne muscular dystrophy pending FDA approval.
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About Cure Rare Disease (CRD)

CRD is a Boston-based nonprofit biotechnology company founded by Rich Horgan, Blavatnik Life Science Entrepreneur-in-Residence at Harvard Business School who has witnessed three generations of his family affected by Duchenne muscular dystrophy, a fatal, neuromuscular disease. As anyone who is touched by a fatal, rare disease knows, time is of the essence. To overcome the obstacles inherent in the existing model of drug development, CRD has taken a new approach, forming an unprecedented collaboration with leading researchers and institutions nationwide in genetics, genomics, bioinformatics and biotechnology. Horgan and team are pioneering the development of a new platform for customized therapeutic treatments that can be applied to a range of rare, genetic diseases, and aim to inspire hope for a cure. Learn more at

About Columbus Children’s Foundation (CCF)

CCF’s mission is to ensure equitable and affordable access to the most effective gene therapy solutions for children with ultra-rare genetic diseases. CCF uses a nonprofit model to partner and accelerate gene therapy treatments for programs with very small patient populations that are often overlooked because the economics don’t add up for those using a traditional commercial development model. Through strategic partnerships with other foundations, industry, patient groups and philanthropy; CCF aims to bring the scientific and funding resources together on behalf of the many ultra-rare diseases to accelerate hope and transform the lives of those fighting these devastating diseases. Learn more at


Nina Pfister