Viralgen Scales up to Produce Gene Therapies with AskBio’s Technology

Viralgen, a Spanish contract development and manufacturing organization (CDMO) half owned by AskBio of Research Triangle Park, has gained vital regulatory status in Europe for producing gene therapies using AskBio’s manufacturing process. 

Viralgen received Current Good Manufacturing Practices (cGMP) compliance accreditation and Pharmaceutical Laboratory authorization by the Spanish Agency for Medicines and Health Products and certification from the European Medicines Agency.

Adherence to the cGMP regulations ensures the proper quality, monitoring, safety, efficacy and information accuracy of medicines and health products for manufacturing processes and facilities.

"We have gone from a bare concrete floor to a fully certified, world-class facility in less than two years,” said Javier García, Viralgen’s chief executive officer. “The quality of our facility, scaled-up manufacturing processes and this certification underscore our commitment to helping our clients develop safer and more effective therapies for genetic disease.”

Making AAV therapies with AskBio's Pro10 manufacturing process

Viralgen specializes in developing, validating and manufacturing adeno-associated virus (AAV) gene therapies by licensing AskBio’s proprietary cell line manufacturing process known as Pro10. Pro10 allows for greater speed, scale, yield and accuracy of AAV therapeutics, according to the company.

“We have an amazing facility and a highly skilled team focused on delivering technology that produces higher yields of rAAV vectors, ultimately contributing to lowering the cost of potentially life-saving therapeutics for patients in need,” García said.

Viralgen’s current production capacity includes: 

• Three independent, state-of-the-art cGMP production suites for manufacturing three different products simultaneously
• 50L scale to supply toxicology/biodistribution studies with full support for research, toxicology and GMP production in parallel
• 50L, 250L and 500L single-use stirred-tank bioreactors for culture of suspension cells with a total capacity of 1,250 liters of dedicated AAV production
• In-house quality control labs for critical release assays.

García said Viralgen plans to expand its current footprint with support for up to 2000L scale for commercial use by the end of 2021.

"Viralgen is a focused and highly capable organization with an unparalleled depth of resources devoted to being the world’s best AAV CDMO," said Sheila Mikhail, chief executive officer and co-founder of AskBio. "Viralgen delivers its clients a superior level of quality to ensure safe, reliable AAV vectors for clinical use that are commercially ready for large-scale production. Together, we look forward to solving many of the gene therapy cost and scale issues that face our industry today."

Jude Samulski, Ph.D., co-founder of AskBio and a pioneer in AAV gene therapeutics, said Viralgen’s production capacity is critical to making gene therapeutics “accessible without limits.”

Samulski's AAV technology is leading gene therapy globally

AAV technology uses engineered virus nanoparticles as vectors to insert corrective DNA into targeted cells in diseased patients. AskBio and several of its spinout companies and partners are developing AAV gene therapies for treating serious genetic diseases including Pompe, limb girdle muscular dystrophy, cystic fibrosis, myotonic muscular dystrophy, Huntington’s disease, hemophilia and Duchenne muscular dystrophy.

AskBio was founded in 2001 as a gene therapy platform company. Earlier this year, the privately held company raised $225 million in venture capital and another $10 million from founders and board members. The funding was the largest single round of venture capital financing ever for a North Carolina bioscience company.

The success of AskBio and similar companies in North Carolina has helped make the state a global leader in gene therapy research and production.

Viralgen, based in San Sebastián, Spain, was founded in 2017 as a joint venture between AskBio and Columbus Venture Partners, a Spanish venture capital group.