Asklepios BioPharmaceutical (AskBio), a Chapel Hill, North Carolina-based developer and manufacturer of Adeno-Associated Virus (AAV) gene therapy therapeutics for underserved patient populations with rare and untreatable genetic disorders, has raised $235 million in funding to accelerate growth. TPG Capital and Vida Ventures will gain a minority stake in the company for the investment.

And as part of the transaction, AskBio’s founders and board members are co-investing $10 million. This investment round provides AskBio with additional capital and resources to help advance and expand clinical trials and enhance its manufacturing capabilities and capacity.

Founded by Jude Samulski, Ph.D. (first scientist to clone AAV), Dr. Xiao Xiao (first to develop a miniaturized dystrophin gene enabling the advancement of gene therapy for Duchenne Muscular Dystrophy), and CEO Sheila Mikhail (accomplished life sciences executive) in 2001, AskBio was formed in the Gene Therapy Center at the University of North Carolina at Chapel Hill.

“It’s humbling to imagine that my Ph.D. project several decades ago has ushered in a new generation of transformative therapies that will impact so many lives,” said Samulski. “I am excited to partner with TPG and Vida to catalyze the next generation of gene therapy innovation and translate discovery into life-changing therapeutics.”

AskBio is known for offering proprietary AAV technology, superior manufacturing capabilities, and a robust pipeline of in-house clinical programs. The company has more than 500 patents in areas like AAV production, chimeric vectors, and self-complementary DNA. And AskBio’s engineered viral vectors are able to target particular tissues, de-target other tissues, and minimize the effects of neutralizing antibodies.

“With the funding from TPG and Vida, we will be able to accelerate our development of a broad range of transformative therapies for those affected by serious and oftentimes incurable genetic diseases,” added Mikhail. “We look forward to advancing our approaches for repeat administration and avoidance of neutralizing antibodies into the clinic to maximize the number of patients who benefit from AAV therapies.”

Gene therapy is considered one of the most active areas of pharmaceutical R&D. And AAV has emerged as an efficient and versatile vehicle for delivering corrective genes inside of the body in recent years.

AskBio also successfully partnered with two companies for its programs. This includes Bamboo Therapeutics for a therapy targeting Duchenne Muscular Dystrophy and other rare diseases. And another program with Chatham Therapeutics with a focus on Hemophilia A and B. These platforms were sold to Pfizer and Baxter (programs now owned by Takeda), respectively.

“We are thrilled to partner with Jude, Sheila, and the entire AskBio team to support the development of therapies for patients suffering from historically untreatable diseases,” explained TPG Capital partner John Schilling, M.D.. “AskBio is a very special company, operating in one of the most innovative and strategically important sectors of the pharmaceutical industry today. In addition to its leading clinical programs in several therapeutic areas, AskBio has a broad base of technologies that we believe can support quantum leaps in therapies for many partners in the market. We are honored to have the opportunity to work with AskBio in the next leg of this journey.”

Another major milestone AskBio achieved is that it recently advanced a therapeutic for Pompe Disease into the clinic based on the work by Dwight Koeberl, M.D., Ph.D., and Priya Kishnani, M.D., M.B.B.S., at Duke University.

“At Vida, we are driven to find innovative companies with disruptive technologies that have the potential to transform how we combat disease,” explained Vida Ventures co-founder and senior managing director Fred Cohen, M.D., Ph.D. “Jude’s acclaimed research in AAV and scientific vision for AskBio, along with the leadership of Sheila and the research expertise of the larger team, exemplify what’s possible when passionate scientists and advocates demand more than conventional therapies.”