Medgenics & CHOP Collaborate
News Nov 22, 2014
Medgenics, Inc. has announced a major research collaboration with The Children's Hospital of Philadelphia (CHOP) focused on pediatric rare and orphan genetic diseases. The goal of the collaboration is to accelerate the development of transformational new therapies for these underserved patients.
The research collaboration will leverage the unique strengths of both organizations. The biobank at CHOP's Center for Applied Genomics (CAG) is home to one of the largest biorepositories of pediatric genetic data in the world.
Access to the rich genetic information in the CAG biobank will uniquely enable CHOP and Medgenics researchers to work rapidly and efficiently to identify new rare and orphan disease targets and accelerate the development of novel therapies into clinical stage programs.
"CHOP created its Center for Applied Genomics, led by Hakon Hakonarson, M.D., Ph.D., to better understand the genetic basis for disease, and we continue to broaden this vision through important collaborations such as this one with Medgenics," said Philip R. Johnson, M.D., Chief Scientific Officer and Executive Vice President at CHOP. "With our pediatric biobank, we bring to this collaboration access to one of the world's largest libraries of novel targets and insights into rare genetic diseases affecting children everywhere. We hope that, with access to our robust pipeline of peptide and protein targets, Medgenics can develop treatments for these rare and orphan diseases that are tailored towards treating the underlying causes of these diseases."
"The mission of Medgenics is to rapidly deliver transformational therapies to those suffering from serious rare and orphan diseases around the world, especially sick children. With our new collaboration with CHOP and their impressive scientific, genomic and clinical resources we will focus our efforts and technology to translate the latest data and insights into new treatments," stated Garry Neil, MD, Chief Scientific Officer of Medgenics. "We believe that our TARGT (Transduced Autologous Regenerative Gene Therapy) technology platform allows us to safely and flexibly harness the power of gene therapy, and our team is anxious to begin our new collaboration with CHOP."
Under the terms of the agreements, Medgenics will pay $5 million to CHOP. Medgenics will have an exclusive license to use the rare and orphan disease samples at the CAG biobank for the purpose of developing and commercializing therapeutic treatments and diagnostic targets for rare and orphan diseases. CHOP will also receive milestone payments and low single-digit royalties on future sales from products developed from the collaboration, and Medgenics will sponsor further research at CHOP on rare and orphan diseases.
"Approximately 10 percent of all rare and orphan disease patients in North America come through CHOP. This will enable Medgenics and CHOP opportunities to link diseases and their genetic abnormalities with potential new treatment paradigms," commented Steven M. Altschuler, M.D., Chief Executive Officer of The Children's Hospital of Philadelphia.
"We are very excited to be launching this unique collaboration with an institution as renowned as CHOP," stated Michael Cola, President and Chief Executive Officer of Medgenics. "Through this effort, we are building a scientific and clinical foundation for Medgenics to become a fully integrated ex vivo gene therapy company, enabling us to develop treatments for patients suffering from so many rare and orphan diseases."
In a new study in cells, University of Illinois researchers have adapted CRISPR gene-editing technology to cause the cell’s internal machinery to skip over a small portion of a gene when transcribing it into a template for protein building. This gives researchers a way not only to eliminate a mutated gene sequence, but to influence how the gene is expressed and regulated.