Columbus Children’s Foundation, a non-profit organization with a mission to bring gene therapeutics to children with ultra-rare genetic diseases, has received an in-kind donation from Viralgen, an AAV gene therapy GMP manufacturer. The donation provides significant capacity for validating and manufacturing adeno-associated virus (AAV) gene therapies to ensure that an underserved population of children who suffer from ultra-rare genetic disorders will receive what are often life-saving treatments.
 
“We can’t thank Viralgen enough for their exceptionally generous and valuable contribution,” said Laura Brod Hameed, the foundation’s executive director. “This donation will help us support treatments that have the promise and power to transform the lives of children in need. Manufacturing often costs millions, and Viralgen’s contribution of capacity will help us with our mission to accelerate treatments for kids who are battling not only a life-threatening disease, but also time.”
 
The high cost of manufacturing AAV vectors often limits the ability of researchers conducting small-batch studies, such as those for ultra-rare conditions. The contribution will help the foundation provide gene therapy research organizations with access to the AAV vectors they need for their studies to validate therapeutics and, ultimately, get into the clinic to provide treatments for kids who desperately need them.  
 
“We are very honored to be a part of the foundation’s work and do our part to make a difference in the lives of these children and their families,” said Javier García, chief executive officer, Viralgen. “With this donation, we are committing manufacturing resources for potentially curative therapeutics that would otherwise not be commercially viable in gene therapy today. It is so heartwarming to see the foundation expand and accelerate equitable and more affordable access to therapeutics to treat children around the globe with these ultra-rare diseases.”
 
The foundation is a unique non-profit biotechnology organization with a specific focus on accelerating access to effective gene therapy treatments for children with ultra-rare genetic diseases. In addition, the foundation provides access to its network of scientific experts, breakthrough technology and manufacturing resources.
 
“The child populations affected by ultra-rare genetic conditions are often too small for large drug companies to justify the investment necessary to make these therapeutics. So, when cures are clinically possible, but not commercially viable, the foundation steps in,” said Sheila Mikhail, a foundation trustee and chief executive officer of AskBio, a gene therapy platform company. “Overcoming the challenge to deliver curative therapies to children who are in desperate need is at the heart of our collective mission.”
 
Along with funding development for gene therapies for ultra-rare conditions, the foundation also provides direct financial resources to patients and their families, providing equitable access to potentially life-saving clinical trials no matter a family’s financial means or locational barriers.