North Carolina-based Asklepios BioPharmaceutical (AskBio), a gene therapy company focused on rare genetic diseases, secured a $235 million investment from Vida Ventures, a venture capital firm backed by Arie Belldegrun, the former chief executive officer of Kite Pharma, and TPG Capital.

The investment, which includes $225 million from Vida Ventures and TPG, as well as $10 million from AskBio’s founders and board members, is earmarked to advance and expand clinical trials, enhance its manufacturing capabilities and capacity, and drive long-term growth. AskBio Chief Executive Officer Sheila Mikhail said the funding will enable to company to accelerate the development of a broad range of therapies for patients impacted by devastating genetic diseases.

Founded in 2001 by Jude Samulski, the first scientist to clone AAV, AskBio recently advanced a gene therapy candidate for Pompe disease into the clinic. In January, the company dosed the first patient with a gene therapy treatment Actus-101 in the Phase I/II trial. Actus-101 is an AAV gene therapy that has been granted Fast Track designation from the U.S. Food and Drug Administration for the treatment of patients with Pompe disease. The genetic disease is caused by a partial or complete deficiency of acid-alpha glucosidase (GAA). The disease is associated with high morbidity and often leads to premature death.

In addition to the Pompe disease therapeutic, AskBio is also developing therapeutics for heart failure, limb-girdle 2i, myotonic dystrophy, and several other muscular and neurological disorders.

AskBio is also the company that initially developed the gene therapy for spinal muscular atrophy that Illinois-based AveXis, a subsidiary of Novartis, is close to securing regulatory approval for in the United States. AskBio received an upfront payment and is eligible for milestone payments and royalties based on AveXis progress and possible commercialization.

The company’s Pro10 AAV manufacturing process has become something of a standard across the industry. The platform is used by multiple companies, including Pfizer, Takeda and Viralgen Vector Core SA. The company holds over 500 patents in areas such as AAV production, chimeric vectors, and self-complementary DNA.

Samulski, president and chief scientific officer of AskBio, said it is humbling that his Ph.D. project from several decades ago has “ushered in a new generation of transformative therapies that will impact so many lives.” He added he was excited about the financing provided by Vida Ventures and TPG. He said the support will “catalyze the next generation of gene therapy innovation and translate discovery into life changing therapeutics.”

Fred Cohen, a co-founder and senior managing director at Vida Ventures, said Samulski’s “acclaimed research in AAV and scientific vision for AskBio” exemplify what is possible when scientists drive to create new therapies that go beyond conventional medicines. Cohen said Vida is driven to support companies that develop such disruptive technologies like AAV, which have the potential to transform the way disease is treated.

“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,” John Schilling, a partner at TPG said in a statement.



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