AskBio gets $235 million in gene therapy support

Posted by on Apr 12, 2019

RESEARCH TRIANGLE PARK, N.C. – Asklepios BioPharmaceutical, Inc (AskBio), a platform company playing a key role in North Carolina’s global gene therapy leadership, got a $235 million vote of confidence this week.

The developer and manufacturer of Adeno-Associated Virus (AAV) gene therapies for underserved patient populations with rare and generally untreatable genetic disorders, announced a $225 million investment from TPG Capital and Vida Ventures in exchange for a minority stake in the company. As part of the transaction, AskBio’s founders and board members are co-investing $10 million.

AskBio plans to use the money to help pay clinical trial expenses, enhance its manufacturing capabilities and capacity, and drive long-term growth. The participants chose not to disclose other terms of the transaction.

AskBio is the brainchild of three pioneers in the field: Richard Jude Samulski, Ph.D. president and chief scientific officer; Xiao Xiao, who was the first to develop a miniaturized dystrophin gene enabling the advancement of gene therapy for Duchenne Muscular Dystrophy; and Sheila Mikhail, J.D., a talented life science business leader who serves as chairman and chief executive officer. Samulski, the first scientist to clone AAV, was recruited to the University of North Carolina School of Medicine from the University of Pittsburgh in 1993 with the help of about $250,000 in grant funding from the North Carolina Biotechnology Center. He directed the Gene Therapy Center at UNC-Chapel Hill for many years.

Several grants and loans from the Biotech Center have supported the development of Samulski’s academic research and commercial technologies respectively. NCBiotech specialists recognized early that gene therapy was poised to become a pharmaceutical R&D game-changer. What’s more, AAV has emerged as a highly promising approach — an efficient, safe, and versatile vehicle for delivering corrective genes inside of the body

Samulski and Mikhail founded AskBio in 2001 to commercialize AAV gene therapies. AskBio has subsequently spun out four gene therapy companies:

  • NanoCor Therapeutics, developing treatments for cardiovascular disease.
  • Chatham Therapeutics, developing treatments for hemophilia. Chatham was sold to Baxter International (now Shire) in 2014 for $70 million.
  • Bamboo Therapeutics, developing treatments for rare neuromuscular diseases. Bamboo was sold to Pfizer in 2016 for $150 million in a deal that could be worth as much as $645 million if certain milestones are met.
  • Actus Therapeutics of Chapel Hill, formed in 2017, started a Phase 1\2 clinical study early in 2019 of its gene therapy in patients with Pompe disease, a rare genetic disorder that often leads to premature death in children and adults.

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