AskBio Receives “Best Venture Funding” Award from Southeast BIO

Recognition highlights $235 million investment fueling growth of company and its industry-leading gene therapy technology platform

Research Triangle Park, N.C., (Nov. 18, 2019) – Asklepios BioPharmaceutical, Inc. (AskBio), a fully integrated Adeno-Associated Virus (AAV) gene therapy platform company focused on providing curative therapeutics for genetic disorders, received the “Best Venture Funding” award from Southeast BIO (SEBIO) at its Investor & Partnering Forum held this month in Miami.

“We selected AskBio from a range of impressive nominations to receive this award for its $235 million round back earlier this year,” said Tina McKeon, chair of SEBIO and partner at the law firm of Kilpatrick Townsend. “This award recognizes companies whose achievements are bringing the Southeast region of the United States into focus as a hub of medical and technological innovation. AskBio is clearly in the forefront of that movement. Congratulations on your success and on this noteworthy recognition.”

Two leading private equity firms, TPG Capital and Vida Ventures, invested a combined $225 million for a minority stake in the company this past April. They were joined by AskBio’s founders and board members who invested an additional $10 million. AskBio is using the additional capital to advance clinical trials, innovate AAV technology, enhance manufacturing capacity, and drive long-term growth.

AskBio was founded in 2001 by Jude Samulski, Ph.D., the first scientist to clone AAV, along with Xiao Xiao, Ph.D., who was the first to develop a miniaturized dystrophin gene enabling the advancement of gene therapy for Duchenne Muscular Dystrophy, and Sheila Mikhail, JD, MBA, an experienced life sciences executive. Since then, the company has built a promising therapeutic pipeline, acquired and sold an array of leading gene therapy companies, and built a gene therapy platform with innovative AAV technology that includes self-complementary AAV and synthetic promoters. AskBio’s platform also includes an expansive library of chimeric capsids, scaled Pro10™ manufacturing, and new delivery methods to accelerate and lower the cost of gene therapy processes and to support its portfolio of curative therapeutics.

“This is a great honor to be acknowledged for our efforts to advance gene therapy,” said Ms. Mikhail, AskBio’s CEO. “Our mission is to develop and distribute curative therapies for the thousands suffering from rare and generally untreatable genetic diseases. Recognition such as this helps to build awareness for the tremendous unmet medical need that exists for those who suffer from devastating genetic diseases.”

Last month, Springboard Enterprises named Ms. Mikhail as its 2019 Northstar Honoree. The award recognizes high-performing female entrepreneurs who are transforming their industries through the discovery, investment and commercialization of breakthrough innovations in technology, science and products that make our lives healthier and more informed.

About AskBio

Founded in 2001, Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, clinical-stage gene therapy platform company dedicated to improving the lives of children and adults with 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. Based in Research Triangle Park, N.C., the company has generated hundreds of proprietary third-generation gene vectors, several of which have entered clinical testing. An early innovator in the space, the company holds more than 500 patents in areas such as AAV production, chimeric vectors, and self-complementary DNA. AskBio maintains a portfolio of clinical programs across a range neurodegenerative and neuromuscular indications with a current pipeline that includes therapeutics for Pompe disease, Limb Girdle Muscular Dystrophy and congestive heart failure as well as out-licensed clinical indications for Hemophilia (Chatham Therapeutics acquired by Takeda) and Duchenne Muscular Dystrophy (Bamboo Therapeutics acquired by Pfizer). For more information, visit

About Southeast BIO

Southeast BIO is a regional non-profit organization that fosters the growth of the life sciences industry in the Southeastern United States through efforts that promote entrepreneurship and bring together the key players active in the development of the industry. Now in its 21st year, the SEBIO Investor & Partnering Forum has built a loyal following, attracting thought leaders and investors from across the country. To date, more than 220 later stage and 180 early stage companies that participated in previous SEBIO Investor & Partnering Forums have raised more than $3.5 billion in public and private funding.