BOSTON,MA (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.
Together the union between CRD and CCF is a leading example of the potential to change the speed of scientific advancement in the field of rare diseases. By establishing this union, CRD and CCF aim to address the economic accessibility issues that nonprofit ultra-rare drug development programs face, especially for scientifically promising therapeutics for n=1 (or a few) patient numbers such as the development of a customized CRISPR therapeutic for the specific mutation of Duchenne muscular dystrophy (DMD) exemplified in this collaboration.
The mission to provide equitable access to customized therapies, including access to AAV, for ultra-rare conditions is the foundation of CRD. Established by Founder & President Rich Horgan, Cure Rare Disease was designed specifically for individuals like his brother, Terry Horgan and others, who are battling an ultra-rare form of DMD for over two decades with no treatment or cure.
“This collaboration is driving the CRD mission to provide therapeutic access for individuals impacted by ultra rare diseases. We aim to encourage other CDMOs to pivot towards successful unions like this one and be inspired to join with CCF in offering more equitable access to high-cost AAV products and helping us in the battle against rare diseases,” says Founder & President Rich Horgan.
Paying sticker price for AAV is currently cost prohibitive to rolling out customized therapeutics. This partnership between CRD and CCF is driving the CRD vision to be realized, starting with the first-in-man human systemic administration of a customized CRISPR therapeutic for DMD. Manufacturing is one of the most difficult steps of this process and working with an industry leader is helping to close the rare disease divide. Working with CCF and their partners, like VVC, also offers access to strategic support and expertise from leaders within the AAV field, paired with a high-quality product that is being produced for the first custom treatment for DMD pending FDA approval.
“CCF is deeply driven by the urgency to ensure equitable access to the most effective treatments for patients with ultra-rare diseases. We believe no one should be left behind when cures are in reach and are thrilled to partner with organizations like CRD to effectuate that mission,” remarked CCF executive director, Laura Hameed.
Through these partnerships, Cure Rare Disease is establishing an ecosystem that society can use as a potential template to develop therapeutics for ultra-rare diseases including unique patient drug development. Today, with this announcement, we are one step closer to our goal of working towards the treatment of all diseases despite their prevalence.
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 www.cureraredisease.org.
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 www.columbuschildren.org