AskBio is a fully integrated AAV gene therapy company – possessing capsid development, scaled manufacturing, and a portfolio of therapeutic programs.

The company has four competitive advantages 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

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.

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, Duchenne 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.