AskBio and AveXis Finalize License

CHAPEL HILL, N.C.– June 17, 2015 –Asklepios BioPharmaceutical, Inc. (AskBio) and AveXis, Inc. (AveXis) have announced entering into a license agreement, which grants AveXis rights to AskBio’s proprietary self-complementary (SC) technology, also known as Duplex vectors. This technology is utilized in AveXis’ Phase I adeno-associated virus (AAV) gene transfer clinical trial in spinal muscular atrophy (SMA1) patients ( SMA is a hereditary disease that affects motor neurons and causes progressive muscle weakness and ultimately death in the most severe forms of the disease.

Under the terms of the agreement, AveXis will maintain non-exclusive access to the use of AskBio’s duplex vectors to support their SMA-based therapeutic development and commercialization efforts. While specific financial terms remain undisclosed, generally the agreement involves AskBio receiving an upfront payment, future payments once certain development and commercial milestones are achieved, as well as a potential related product royalty.

“AskBio’s duplex vectors have a long-standing proven track record of demonstrating faster onset and higher levels of therapeutic gene expression. As an AAV platform technology, AskBio’s self-complementary technology has been shown to be effective at decreasing dosing requirements. In turn, this reduces manufacturing demands across a broad range of therapeutic areas. This transaction is further validation of the diverse applicability of another one of AskBio’s platform technologies, and represents another opportunity to positively affect patient outcomes. We are pleased to be formally associated with an effort designed to provide potentially therapeutic results for such a vulnerable patient population, and one in such obvious and significant need,” said Jude Samulski, PhD, Vice President of AskBio.

“AveXis is committed to the development of novel treatments for patients with SMA. The self-complementary technology allows for the rapid production of the survival motor neuron (SMN) protein, which is critical for SMA patients where motor neuron dysfunction and death along with muscle weakness progresses rapidly after the initial onset of symptoms. We believe this technology is a key component to the promising data we have seen thus far, and we are pleased to establish this agreement with AskBio,” said Brian K. Kaspar, PhD, Scientific Founder, AveXis.

About Asklepios BioPharmaceutical
AskBio is a privately held biotechnology company engaged in the clinical development of novel gene therapies for a broad diversity of diseases with unmet or under-served patient needs. AskBio is currently developing proprietary gene-delivery technologies for therapeutics, targeting diseases in the heart, CNS, muscle, ocular and liver tissues. For additional information, visit AskBio’s website at

About AveXis
Based in Dallas, Texas, AveXis is a clinical-stage gene therapy platform company establishing unique industry alliances to create innovative treatments for people with unmet medical needs. Spinal muscular atrophy (SMA) is the company’s first focus. For more information regarding AveXis, please visit: