AskBio Gets $235M in Gene Therapy Support

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Asklepios BioPharmaceutical, Inc (AskBio), a Research Triangle Park platform company playing a key role in North Carolina’s global gene therapy leadership, got a $235 million vote of confidence today.

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. 

Richard Jude Samulski, Ph.D. president and chief scientific officer
Richard Jude Samulski, Ph.D. president and CSO.
-- AskBio photos

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.

Pfizer invests in NC's gene therapy growth trajectory

Pfizer has more recently tapped NCBiotech to administer a $4 million fellowship program to continue building the gene therapy expertise already evident in North Carolina. The Pfizer-NCBiotech Distinguished Postdoctoral Fellowship in Gene Therapy (Pfizer-GTF) program is a partnership between Pfizer, Inc. and the North Carolina Biotechnology Center that supports the scientific and professional development of exceptional postdoctoral fellows interested in establishing careers in gene therapy.

Competitive awards fund two-year fellowships that enable early career scientists to conduct research in the laboratories of gene therapy-related investigators at any North Carolina university.

AskBio scientist reviews panel in lab.

AskBio holds more than 500 patents in areas such as AAV production, chimeric vectors, and self-complementary DNA. AskBio’s engineered viral vectors are able to target particular tissues, de-target other tissues, and minimize the effects of neutralizing antibodies.

“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.”

Mikhail added, “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. 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.”

John Schilling, M.D., a partner at TPG Capital, added, “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. 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.”

Therapies for Pompe, heart failure, other muscular and neurological disorders

AskBio 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. The company is also developing therapeutics for heart failure, Limb Girdle 2i, Myotonic Dystrophy, and several other muscular and neurological disorders. AskBio’s AAV manufacturing process, Pro10, currently yields 1018 in 200-liter bioreactors and is recognized within the industry as the gold standard in scale manufacturing. Pro10 is used by Pfizer, Takeda, and Viralgen Vector Core SA. Recently, AskBio formed a partnership with Touchlight Genetics Ltd. for the commercialization of a safer, cheaper, and more-efficient alternative to plasmid DNA.

“At Vida, we are driven to find innovative companies with disruptive technologies that have the potential to transform how we combat disease,” said Fred Cohen, M.D., Ph.D., co-founder and senior managing director at Vida Ventures. “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.”

Some of TPG Capital’s past and current investments in the pharmaceutical industry include Par Pharmaceutical (now known as Endo International), Adare Pharmaceuticals, Amneal Pharmaceuticals, Aptalis, EnvisionRx, Quintiles, and IMS (together, now known as IQVIA). Last year, TPG Capital and Vida partnered to invest in Allogene Therapeutics, Inc (NASDAQ: ALLO). Vida’s recent biotechnology investments include Kronos Bio, Praxis Precision Medicines, Oyster Point Pharma, and Pionyr Immunotherapeutics.

Piper Jaffray served as financial advisor to AskBio in this transaction.

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