BioSpace.com

Biotech and Pharmaceutical
News & Jobs
Search the Site
 
   
Biotechnology and Pharmaceutical Channel Medical Device and Diagnostics Channel Clinical Research Channel BioSpace Collaborative    Job Seekers:  Register | Login          Employers:  Register | Login  

NEWSLETTERS
Free Newsletters
Archive
My Subscriptions

NEWS
News by Subject
News by Disease
News by Date
PLoS
Search News
Post Your News
JoVE

CAREER NETWORK
Job Seeker Login
Most Recent Jobs
Browse Biotech Jobs
Search Jobs
Post Resume
Career Fairs
Career Resources
For Employers

HOTBEDS
Regional News
US & Canada
  Biotech Bay
  Biotech Beach
  Genetown
  Pharm Country
  BioCapital
  BioMidwest
  Bio NC
  BioForest
  Southern Pharm
  BioCanada East
  US Device
Europe
Asia

DIVERSITY

INVESTOR
Market Summary
News
IPOs

PROFILES
Company Profiles

START UPS
Companies
Events

INTELLIGENCE
Research Store

INDUSTRY EVENTS
Biotech Events
Post an Event
RESOURCES
Real Estate
Business Opportunities

 News | News By Subject | News by Disease News By Date | Search News
eNewsletter Signup
Miles
Km80.5

   

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:
http://cordis.europa.eu/projects

For the EC press release about rare diseases research projects (including MEUSIX), go to:
http://europa.eu/rapid/press-release_MEMO-13-148_en.htm

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 www.REGENXbio.com


Contact:
REGENX Biosciences
Vit Vasista
202-785-7438
Email Contact

Digg this    Bookmark with del.icio.us    Add to Newsvine

Technorati:



Read at BioSpace.com

   

ADD TO DEL.ICIO.US    ADD TO DIGG    ADD TO FURL    ADD TO STUMBLEUPON    ADD TO TECHNORATI FAVORITES