SOURCE: ReGenX BioSciences

REGENX BioSciences

March 08, 2012 06:00 ET

REGENX BioSciences and Asklepios BioPharmaceutical Enter Into Agreement for the Development of Treatments for Hemophilia A Using NAV™ rAAV8 Vectors

WASHINGTON, DC--(Marketwire - Mar 8, 2012) - REGENX BioSciences, LLC announces that it has entered into an agreement with Chatham Therapeutics, LLC, an affiliate of Asklepios BioPharmaceutical, Inc. (AskBio), for the development and commercialization of products to treat Hemophilia A using NAV™ rAAV8 vectors.

Under the terms of the agreement, Chatham and REGENX entered into a research collaboration, and REGENX provided Chatham with a non-exclusive license to use REGENX's NAV rAAV8 in Chatham's research and pre-clinical development of therapeutics that deliver DNA, RNA or other sequences for treatment of hemophilia A. The rights granted to Chatham include, but are not limited to, use in methods for protein replacement, modulation of gene expression, as well as gene editing approaches. Additionally, Chatham has the exclusive right to exercise an option for an exclusive worldwide license, with rights to sublicense, to REGENX's NAV rAAV8 vectors for the treatment of Hemophilia A in humans, subject to certain limitations. In return for these rights, REGENX receives payments in the form of up-front and on-going fees and is eligible to receive future payments beginning at the exercise of the license option including up-front and on-going fees, certain milestone fees and royalties on net sales of products incorporating NAV rAAV8. REGENX would also receive a share of any sublicensing revenues.

"We believe that this collaboration and license option with Chatham is important and will enable the development of NAV rAAV8-based gene delivery treatments for hemophilia," said Ken Mills, President and Chief Executive Officer of REGENX. "Chatham's development programs combine the outstanding proprietary technologies and expertise of REGENX and AskBio. This agreement represents the continued interest of REGENX to ensure access to our NAV technology for partners that evidence expertise and commitment to development of novel therapies."

"We are excited to build upon our extensive IP platform with this new license, adding to our preexisting exclusive rights to rAAV8 for the field of treating Hemophilia B. We are on track to advance our rAAV8-based Hemophilia B therapeutic into clinical testing later this year," said Scott McPhee, Vice President at AskBio and Co-Founder of Chatham Therapeutics. "We are confident that the science and synergy supporting these Hemophilia A & B programs will result in efficient clinical development and product approval processes."

Hemophilia A is caused by a deficiency of the clotting factor designated Factor VIII. The main symptom is uncontrolled, often spontaneous bleeding. Internal bleeding into the joints can result in pain, swelling and, if left untreated, can cause permanent damage. The worldwide incidence of hemophilia A is one case per 5,000 male births, translating to 700,000 cases in all, with approximately 17,000 of those cases in the US. A recently published clinical study in the New England Journal of Medicine reported correction in patients with hemophilia B using NAV rAAV8 vectors, demonstrating the utility and safety of the platform.

About REGENX BioSciences, LLC

REGENX BioSciences, LLC is leading the effort to translate promising gene delivery applications into a pipeline of next generation personalized therapies for a range of severe diseases with serious unmet needs. We believe that the NAV™ technology to which we have exclusive rights represents the potential promise of curing the root cause of disease rather than the symptoms. We are committed to establishing best in class standards for our NAV vectors. Our intent is to initially develop treatments for a number of rare, genetic diseases including hypercholesterolemias, the mucopolysaccharidoses, and retinitis pigmentosa and to ensure continuing access to our NAV technology through innovative partnerships, license opportunities, as well as the expansion of our growing team of global collaborators. REGENX holds exclusive rights to a portfolio of over 100 patents and patent applications pertaining to its NAV technology and related applications. Visit to learn more.

About Asklepios BioPharmaceutical, Inc.

Asklepios BioPharmaceutical, Inc. (AskBio) is a private clinical development-stage biotechnology company engaged in the development of novel, gene therapy mediated protein therapies using a proprietary Biological Nano Particle (BNP™) technology platform. BNPs may be used to deliver a broad variety of biological material to a cell, including therapeutic genes, monoclonal antibodies, RNAi, and vaccines, among others. Chatham Therapeutics, LLC is a subsidiary of AskBio focusing on a potential cure for hemophilia, thru the use of the BNP platform technologies. The company is currently engaged in gene therapy based therapeutic programs for both hemophilia A and B and will commence a Phase I/II clinical trial for its Hemophilia B program in 2012. For additional information, visit AskBio's Web site at

Contact Information

  • Contact:
    REGENX BioSciences
    Vit Vasista
    Email Contact

    Chatham Therapeutics & Asklepios BioPharmaceutical
    Jade Samulski
    Email Contact


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