AskBio reels in $235M to ramp up gene therapy clinical trials, manufacturing

Illustration of three DNA helices
Since its founding in 2001, the company has spun out and sold two companies: hemophilia-focused Chatham Therapeutics and Bamboo Therapeutics, which specializes in Duchenne muscular dystrophy and other rare diseases. (Darwin Laganzon)

TPG Capital and Vida Ventures are stumping up $225 million for a minority stake in Asklepios BioPharmaceutical—capital that will join another $10 million from the company’s founders and board members to propel its gene therapy pipeline through development. 

Better known by the less unwieldy moniker AskBio, the company will use the cash to advance and expand clinical trials and boost manufacturing for a pipeline of adeno-associated virus (AAV) gene therapies across neuromuscular, central nervous system and heart diseases. Its most advanced programs are treatments for Pompe disease, a rare genetic disorder, and Parkinson’s disease. 

AskBio was formed in the Gene Therapy Center at the University of North Carolina at Chapel Hill and counts among its co-founders R. Jude Samulski, Ph.D., the first scientist to clone AAV. With its gene therapy platform, AskBio develops drug candidates that it may choose to keep in-house or spin out into a subsidiary. 

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Since its founding in 2001, the company has spun out and sold two companies: hemophilia-focused Chatham Therapeutics and Bamboo Therapeutics, which specializes in Duchenne muscular dystrophy and other rare diseases. Baxter acquired Chatham for $70 million in 2014, while Pfizer snapped up Bamboo for for $645 million two years later. 

RELATED: Pfizer puts Bamboo’s muscular dystrophy gene therapy to the test 

“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 AskBio CEO and co-founder Sheila Mikhail in a statement. “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 is developing its Pompe candidate through its subsidiary, Actus Therapeutics, which it spun out last April. The asset is based on the work of Duke University scientists Dr. Dwight Koeberl and Dr. Priya Kishnani. In addition to its clinical-stage programs, it is also working on several preclinical assets, including those for Huntington disease, epilepsy, heart failure and limb-girdle muscular dystrophy type 2i.

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