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REGENX Biosciences, LLC Consortium Awarded $7.7 Million Grant From EC for the Advancement of Clinical Development Program Using NAV™ rAAV8 Vectors

3/27/2013 10:27:27 AM

WASHINGTON, DC--(Marketwire - March 27, 2013) -

REGENX Biosciences, LLC announces that it has received a grant award from the European Commission's (EC's) Seventh Framework Program as a part of the MEUSIX consortium, a collaboration consisting of eight partners and led by Fondazione Telethon. The funding will enable a multicenter phase 1/2 clinical trial to investigate the safety and efficacy of NAV™ rAAV8-based gene therapy in patients with Mucopolysaccharidosis VI (MPS VI), a rare lysosomal storage disorder.

The EC is providing total funding of EUR 6.0 million ($7.7 million) to the MEUSIX consortium. The European Commission recently announced on Rare Disease Day 2013 that this grant was a part of EUR 144 million ($186 million) of new funding focused on rare disease research projects that will help improve the lives of some of the 30 million Europeans suffering from a rare disease.

"We are pleased the European Commission has awarded this grant that will enable us to participate with Fondazione Telethon and our other partners in a first-in-human study using a NAV rAAV8-based gene delivery treatment for MPS VI," said Ken Mills, President and CEO, REGENX Biosciences. "The results from this clinical trial could have a tremendous impact on the natural history of MPS VI and significantly improve the quality of life of the affected patients. Moreover, the approach may facilitate the development of similar approaches for other inborn errors of metabolism. As the lead commercial partner in the MEUSIX consortium, we are committed to helping accelerate the development of meaningful treatments for MPS VI for patients worldwide."

Mucopolysaccharidosis VI (MPS VI, or Maroteaux-Lamy syndrome) is a rare lysosomal storage disease caused by deficient activity of arylsulfatase B (ARSB). MPS VI is characterized by growth retardation, corneal clouding, cardiac valve disease, organomegaly, and skeletal dysplasia, without central nervous system involvement. Thus, systemic therapies targeting peripheral organs have the potential to fully correct the MPS VI phenotype. Enzyme replacement therapy, the current treatment for MPS VI, requires weekly infusions of a costly enzyme and has limited efficacy on bone and corneal disease. Based on the encouraging preclinical results generated by the members of the MEUSIX consortium, gene therapy based on a single intravascular administration of NAV rAAV8 vectors targeting the liver has the potential to provide a durable source of ARSB. It is estimated that approximately 1,200 patients in developed countries have MPS VI.

For more information about the MEUSIX award and activities, go to:

For the EC press release about rare diseases research projects (including MEUSIX), go to:

About REGENX Biosciences

REGENX Biosciences 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, and 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 ensure continuing access for our NAV technology through innovative partnerships, license opportunities and 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

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