Columbus Children's Foundation receives commitment to accelerate production of curative treatments for children with ultra-rare genetic diseases

Viralgen, a world-leading GMP manufacturer of AAV therapeutics, makes significant contribution in manufacturing capacity to serve children in need

CHAPEL HILL, N.C., Feb. 4, 2020 /PRNewswire/ -- 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, a world-leading 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.

AskBio, ViralGen, and Columbus Children's Foundation come together for children with ultra-rare diseases at a charitable concert at the Prado in Madrid, Spain.

"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 Garcia, Viralgen's CEO. "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 CEO of AskBio, a leading 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.

We invite other individuals and organizations to join us in supporting the foundation's work to expand access to genetic medicine for children who face life-threatening genetic diseases. To learn more about Columbus Children's Foundation or to donate, visit columbuschildren.org.

About Columbus Children's Foundation
Founded in 2017, Columbus Children's Foundation is a 501(c)3 non-profit in Chapel Hill, N.C. Its mission is to help children diagnosed with ultra-rare genetic diseases. Columbus Children's Foundation is focused on ultra-rare disease that have extremely small populations. Because the pharmaceutical industry tends to focus on more common illnesses with greater commercial potential, orphan diseases are often overlooked, prompting the need for funding from outside the industry. Columbus Children's Foundation also helps ensure that children with such disorders can participate in clinical trials or receive therapy if their families can't afford travel and related costs. For more information, visit our website or call (919) 726-8040.

About Viralgen
Viralgen is a world-class AAV cGMP clinical and commercial manufacturer, founded in 2017 as a joint venture between AskBio and the Spanish venture capital group, Columbus Venture Partners. Viralgen licenses AskBio's Pro10™ cell line technology that allows for greater scale, higher yields and increased accuracy of AAV therapeutics. Based at the Miramon Parke in San Sebastián, Spain, Viralgen is a CDMO (Contract Development and Manufacturing Organization) that produces AAV gene therapy treatments to enable pharmaceutical and biotechnology companies to accelerate the delivery of novel treatments to improve patient lives. Learn more about Viralgen at viralgen.com.

About AskBio
Founded in 2001, Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, clinical-stage gene therapy company dedicated to improving the lives of children and adults with genetic disorders. AskBio's gene therapy platform includes an industry-leading proprietary cell line manufacturing process called Pro10™ and an extensive AAV capsid and promoter library. Based in Research Triangle Park, North Carolina, the company has generated hundreds of proprietary third-generation AAV capsids and promoters, several of which have entered clinical testing. An early innovator in the space, AskBio holds more than 500 patents in areas such as AAV production and chimeric and self-complementary capsids. AskBio maintains a portfolio of clinical programs across a range of neurodegenerative and neuromuscular indications with a current clinical pipeline that includes therapeutics for Pompe disease, limb-girdle muscular dystrophy 2i/9R and congestive heart failure, as well as out-licensed clinical indications for hemophilia (Chatham Therapeutics acquired by Takeda) and Duchenne muscular dystrophy (Bamboo Therapeutics acquired by Pfizer). Learn more at https://www.askbio.com or follow us on LinkedIn.

Media Contact:
Laura Brod Hameed
Executive Director
lhameed@columbuschildren.org
612-437-8836

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SOURCE Columbus Children's Foundation

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