Gene Therapy Developer AskBio Secures $235 Million Investment

April 12, 2019

Asklepios BioPharmaceutical (AskBio) said TPG Capital and Vida Ventures have taken a minority stake in the company for $235 million to advance and expand its clinical trials, enhance its manufacturing capabilities, and drive long-term growth.

As part of the transaction, AskBio’s founders and board members are co-investing $10 million.

“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,” said Sheila Mikhail, CEO and Co-Founder of AskBio. “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.”

AskBio was founded in 2001 by Jude Samulski, the first scientist to clone AAV, along with Xiao Xiao, who was the first to develop a miniaturized dystrophin gene enabling the advancement of gene therapy for Duchenne muscular dystrophy, and company CEO Sheila Mikhail, an accomplished life sciences executive. The company was formed in the Gene Therapy Center at the University of North Carolina at Chapel Hill.

AskBio offers proprietary AAV technology, best-in-class manufacturing capabilities, and a robust pipeline of in-house clinical programs. An early innovator in the space, the company holds over 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.

AskBio recently advanced a therapeutic for Pompe disease into the clinic based on the work by Dwight Koeberl and Priya Kishnani at Duke University. The company is also developing therapeutics for heart failure, Limb Girdle 2i, myotonic dystrophy, and several other muscular and neurological disorders.

Photo: Sheila Mikhail, CEO and Co-Founder of AskBio

Author: Rare Daily Staff

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