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Rare Diseases: Medgenics And Children’s Hospital Of Philadelphia Collaboration

November 13, 2014

Medgenics and the Children’s Hospital of Philadelphia (CHOP) announce on November 12th a research collaboration focusing on pediatric rare and orphan genetic diseases. About 10% of rare disease patients in North America come through CHOP.

The partnership involves use of the biobank at CHOP’s Center for Applied Genomics (CAG). The mission of CAG:

“ is to develop new and better ways to diagnose and treat children affected by complex medical disorders … to discover genetic causes for the most prevalent diseases of childhood … objective is to generate new diagnostic tests and to guide physicians to the most appropriate therapies … have large-scale access to state-of the-art high-throughput genotyping technology …”.

CAG’s biobank is one of the largest biorepositories of pediatric genetic data in the world. CAG’s biobank will provide access to genetic information that will help CHOP and Medgenics researchers to identify new rare disease targets that will help to accelerate development of therapies into clinical stage programs.

Medgenics is providing their TARGT (Transduced Autologous Regenerative Gene Therapy) technology platform that will provide gene therapy for the collaboration with CHOP. Under the new collaboration:

•   Medgenics pays $5 million to CHOP

•   Medgenics will have exclusive license to use rare disease CAG biobank samples for developing/commercializing treatments and diagnostic targets

•   CHOP to receive milestone payments & royalties on future sales from products developed

•   Medgenics will sponsor additional research at CHOP on rare diseases.

Please Note: “Pathologist with Microscope” courtesy of Bill Branson (Photographer) from the National Cancer Institute (NCI). [Public domain in the US].

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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