AskBio – Leading Gene Therapy Company Announces $235 Million Investment

Asklepios BioPharmaceutical, Inc (AskBio), announced today to have raised $235m in funding to augment and accelerate its business. TPG Capital and Vida Ventures are investing $225m for a minority stake in the company. As part of the transaction, AskBio’s founders and board members are co-investing $10m. The company intends to use the funds to advance and expand clinical trials, enhance its manufacturing capabilities and capacity, and drive long-term growth.

Founded: 2001
Total Investment:
$235m
Investors:

  • TPG Capital and Vida Ventures ($225m)
  • Company founders and Board members ($10m)

Purpose of fundraising:

  • Advance and expand clinical trials
  • Enhance its manufacturing capabilities and capacity
  • Drive long-term growth

Asklepios BioPharmaceutical, Inc (AskBio), a developer and manufacturer of Adeno-Associated Virus (AAV) gene therapy therapeutics for underserved patient populations with rare and generally untreatable genetic disorders, announced today $235 million in funding  TPG Capital and Vida Ventures are investing $225 million for a minority stake in the company. As part of the transaction, AskBio’s founders and board members are co-investing $10 million. The investment provides AskBio with additional capital to help advance and expand clinical trials, enhance its manufacturing capabilities and capacity, and drive long-term growth. Additional terms of the transaction were not disclosed.

AskBio was founded in 2001 by Dr. Jude Samulski, Ph.D., the first scientist to clone AAV, along with Dr. 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. 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.

READ  Top 10 Biotechnology Companies in the USA

Dr. Jude Samulski, Ph.D., President and Chief Scientific Officer of AskBio says,

“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. 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.”

Sheila Mikhail, CEO, and Co-Founder of AskBio says,

“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., Partner at TPG Capital says,

“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.”

About AskBio

Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held AAV gene therapy based company founded in 2001 to advance the work of Dr. Richard Jude Samulski, former Director of the Gene Therapy Center at the University of North Carolina (UNC) from the lab into the clinic. Dr. Samulski was the first to clone AAV for therapeutic purposes. Since AskBio’s inception almost two decades ago, Dr. Samulski’s efforts have joined those of a group of widely acclaimed researchers and clinicians.

READ  Roundup: Monthly Biotech Updates, February 2019

Four competitive advantages of AskBio, relative to other companies in the AAV space:

  • Next Generation AAV Technology
  • Broad Patents and Intellectual Property Portfolio
  • Scaled-Up Manufacturing
  • A Broad Portfolio of Therapeutic Programs

The company’s history has been largely shaped by parents who turned to gene therapy as a treatment option for diseases for which conventional medicine had not provided any solutions. In fact, the company’s name is based on the Greek mythology character, Asclepius, who cured incurable diseases, and is often recognized as the symbol of modern medicine. Through the advocacy of these parents, AskBio has worked on a range of diseases such as hemophilia, Duchene Muscular Dystrophy (DMD), Giant Axonal Neuropathy (GAN), and late-stage heart failure (initially focused on the cardiomyopathy experienced by DMD patients).

In the early days of the company, little institutional funding was available for gene therapy, which was seen as a young and unproven field. AskBio was able to advance its work through the funding of its founding parents (who in some cases raised funds through bake sales and other grassroots strategies) and through patient advocacy groups such as the Muscular Dystrophy Association (the MDA). AskBio received the first grant made by the MDA to a for-profit entity. AskBio has met each of the founding parent’s hopes through the successful advancement of an AAV therapeutic for each of the diseases suffered by these parents’ children.

Today, AskBio have 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.

READ  3 Things That Will Prove Why Bio-robotics Will Be The Future of Medical Science