Leading gene therapy company  announces $235 million investment to accelerate growth

Research Triangle Park, NC; Fort Worth, Texas; San  Francisco; and Boston – April 11, 2019 – 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  to augment and accelerate its business. 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.

“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,” said Dr. Jude Samulski, Ph.D., President and Chief  Scientific Officer of AskBio. “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.”

“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.”

Gene therapy has been one of the most active areas of  pharmaceutical R&D, and in recent years, AAV has emerged as an efficient,  safe, and versatile vehicle for delivering corrective genes inside of the body.  AskBio has successfully partnered with companies on two of its programs: Bamboo  Therapeutics, a therapy targeting Duchenne Muscular Dystrophy and other rare  diseases, and Chatham Therapeutics, which focuses on Hemophilia A and B. The  platforms were sold to Pfizer and Baxter (programs now owned by Takeda),  respectively.

“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,” said John Schilling, M.D.,  Partner at TPG Capital. “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.”

AskBio recently 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. 

“At Vida, we are driven to find innovative companies with  disruptive technologies that have the potential to transform how we combat  disease,” said Fred Cohen, M.D., Ph.D., Co-Founder and Senior Managing Director  at Vida Ventures. “Jude’s acclaimed research in AAV and scientific vision for  AskBio, along with the leadership of Sheila and the research expertise of the  larger team, exemplify what’s possible when passionate scientists and advocates  demand more than conventional therapies.”

Some of TPG Capital’s past and current investments in the  pharmaceutical industry include Par Pharmaceutical (now known as Endo  International), Adare Pharmaceuticals, Amneal Pharmaceuticals, Aptalis,  EnvisionRx, Quintiles, and IMS (together, now known as IQVIA). Last year, TPG  Capital and Vida partnered to invest in Allogene Therapeutics, Inc (NASDAQ:  ALLO). Vida’s recent biotechnology investments include Kronos Bio, Praxis  Precision Medicines, Oyster Point Pharma, and Pionyr Immunotherapeutics.

Piper Jaffray served as financial advisor to AskBio in  connection with this transaction.

About AskBio

Asklepios BioPharmaceutical, Inc. (AskBio) is a privately  held, gene therapy platform company dedicated to improving the lives of  children and adults with rare genetic disorders. AskBio’s gene therapy platform  includes an industry-leading proprietary cell line manufacturing process known  as Pro10 and an extensive AAV capsid library. The company has generated  hundreds of proprietary third generation gene vectors that have entered  clinical testing, and maintains a portfolio of clinical programs across a range  of indications including Pompe, Limb Girdle Muscular Dystrophy, Cystic  Fibrosis, Myotonic Muscular Dystrophy, Huntington’s, Hemophilia (Chatham  Therapeutic/Shire) and Duchenne Muscular Dystrophy (Bamboo  Therapeutics/Pfizer). For more information, visit www.askbio.com.

About TPG

TPG is a leading global alternative asset firm founded in  1992 with more than $104 billion of assets under management and offices in  Austin, Beijing, Boston, Dallas, Fort Worth, Hong Kong, Houston, London,  Luxembourg, Melbourne, Moscow, Mumbai, New York, San Francisco, Seoul, and  Singapore. TPG's investment platforms are across a wide range of asset classes,  including private equity, growth venture, real estate, credit, and public  equity. TPG aims to build dynamic products and options for its investors while  also instituting discipline and operational excellence across the investment  strategy and performance of its portfolio. For more information, visit www.tpg.com

About Vida Ventures

Vida Ventures is a next-generation life sciences investment  firm founded in 2017 by a group of scientists, physicians, entrepreneurs and  investors passionate about building and funding breakthroughs in biomedicine.  Together they form an independent, bold investment group bound together by a  simple word – life. Its mission is to bring science to life and advance  transformative biomedical innovations that have the potential to make a  meaningful difference for patients. Vida has a bicoastal presence and currently  manages approximately $355 million. For more information on Vida Ventures,  please visit www.vidaventures.com, on  LinkedIn or follow on Twitter @Vida_Ventures.

Media Contacts

TPG Capital
Luke Barrett
415-743-1550
media@tpg.com