Hemophilia. Duchenne muscular dystrophy. Epilepsy. Alzheimer’s disease. Diabetic retinopathy. Age-related macular degeneration. Osteoarthritis.

These are just some of the diseases that Chapel Hill-based Asklepios BioPharmaceutical (AskBio) is working to alleviate through the development and delivery of novel, intracellular protein therapeutics using its cutting-edge Biological Nano Particle (BNP) technology.

This gene-transfer technology is used to deliver a broad variety of biological material, including therapeutic genes, RNA interference (RNAi) – a biological process in which RNA molecules inhibit gene expression – and vaccines, among others, to specific tissues.

“Think of our BNP gene transfer vectors like a tiny FedEx truck delivering corrective DNA to specific targeted tissues,” explains Charlie Turner, a partner in Technology Commercialization Group, a business development consultant serving AskBio and other RTP-based biotechnology companies.

“Once delivered, the DNA within the patient’s cell generates therapeutic proteins to correct a disease. This gene therapy ‘truck’ is AskBio’s proprietary delivery system, or platform technology.”

Business model creates spinout companies

AskBio’s business model creates spinout companies and licenses its proprietary platform technology for specific diseases. For example, hemophilia was licensed into Chatham Therapeutics, as a spinout. Partially owned by AskBio, together with other investors and inventors, Chatham caught the attention of Baxter International Inc., which purchased it for $70 million up front, with much larger undisclosed potential over the next decade as products advance.

To date, AskBio’s gene therapy technology has been licensed by a number of the major biotechnology and large pharmaceutical companies world-wide including Merck, GSK, Pfizer, Baxter, Sanofi-Genzyme, and Medtronic, among many others.



AskBio was founded by R. Jude Samulski, Ph.D., a professor recruited to the University of North Carolina at Chapel Hill School of Medicine with the help of a $430,000 NCBiotech Faculty Recruitment Grant in 1993. Four other spinouts are partially owned by the company. Each has a therapeutic focus, including orphan and underserved conditions such as Duchenne muscular dystrophy. They include:

  • Forsight Pharmaceuticals (ocular)
  • NanoCor Therapeutics (cardiovascular)
  • Asklepios DMD (Duchenne muscular dystrophy)
  • Provectra (central nervous system)

Each spinout has its own management, with responsibility for funding and business development.

In AskBio’s early days, its sustainability was a concern due to the financial challenges related to the economic biotech bubble, the rapid decline of the stock market, and consequentially a financing vacuum for start-up biotechs.

However the North Carolina Biotechnology Center, along with key long-term angel investors, recognized the potential in AskBio and provided crucial funding.

AskBio Delivers; NCBiotech Helps

“NCBiotech’s grants and loans were important in assisting with maintaining our early stage R&D momentum,” said Turner. “The funding came at critical times – it was so difficult to get early stage funding – especially in the risky area of gene therapy.”

NCBiotech funding that has contributed to AskBio’s evolution includes:

  • The $430,000 Faculty Recruitment Grant bringing Samulski to North Carolina.
  • A $15,000 start-up loan in 2004 which at the time was called a Business Development Award. Today it’s been replaced by the Company Inception Loan.
  • A $150,000 Small Business Research Loan in 2005 to help select the best use of therapy for congestive heart failure.
  • A $100,000 Collaborative Funding Grant (CFG) in 2007 to advance the development of a potential heart failure therapy.
  • A $350,000 Small Business Research Loan in 2009 to support research on osteoarthritis.
  • A $100,000 CFG in 2013 for hemophilia A gene therapy applications.

In addition, AskBio received a 2004 Venture Philanthropic grant of $1.6 million from the Muscular Dystrophy Association (MDA) providing preclinical and clinical research and development financial support. At the time this was the largest award to a private company in MDA’s 54-year history.

Early NCBiotech funding proved crucial

Because of the early and timely funding from NCBiotech, AskBio maintained traction — and attracted additional significant grant and investment funding. It paved the way for innovative and curative therapies that have the potential to improve patient quality of life, reduce treatment time, and save hundreds of thousands of dollars per patient annually.

“Early stage funding is imperative if we want to see real innovation in North Carolina,” says Turner. “Our competitors in Boston, San Francisco, New York and San Diego have tremendous funding resources at their disposal. Innovative companies that start in North Carolina are moving to these places because they can get the money they need.

“We (North Carolina biotechnology companies) are competing with these hotbeds of capital, and it’s challenging to do without a diversity of funding opportunities. The more we can get early stage funding to help companies advance breakthrough, change-the-world technologies and products, the more of them will stay in North Carolina and succeed.

“We are a great example of how NCBiotech’s funding assisted us getting started and continue the important work we’re doing. Success breeds success.”

(C) NC Biotechnology Center