AAV gene therapy company AskBio secures $235m investment

Asklepios BioPharmaceutical (AskBio) has secured an investment of $235m to accelerate its business of developing and manufacturing Adeno-Associated Virus (AAV) gene therapies for rare and generally untreatable genetic disorders.

The investment in AskBio includes an amount of $225m committed by global alternative asset firm TPG Capital and life sciences investment firm Vida Ventures in exchange of a minority stake in the North Carolina-based AAV gene therapy company.

AskBio’s founders and board members are investing $10m in the company as well. The new capital is expected to help advance and expand clinical trials of the company, while improving its manufacturing capabilities and capacity, and helping it generate long-term growth.

AskBio CEO and co-founder Sheila Mikhail said: “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.”

Founded in 2001, AskBio has more than 500 patents in areas like AAV production, self-complementary DNA and chimeric vectors. The company’s engineered viral vectors are said to be capable of targeting particular tissues, de-target other tissues, and minimize the impact of neutralizing antibodies.

Recently, the company advanced a gene therapy for Pompe Disease into clinic development. In addition, it is developing therapies for heart failure, Myotonic Dystrophy, Limb Girdle 2i along with other muscular and neurological disorders.

The company’s gene therapy platform includes a cell line manufacturing process called Pro10 in addition to having an extensive AAV capsid library. Pro10 is used by Pfizer, Viralgen, Takeda and Vector Core, said AskBio, which recently entered into a partnership with Touchlight Genetics for the commercialization of a safer, more efficient and cheaper alternative to plasmid DNA.

TPG Capital partner John Schilling said: “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.”

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