Medgenics, CHOP enter rare and orphan disease research collaboration

The collaboration, which will leverage the strengths of both organizations, is aimed at accelerating the development of transformational new therapies for these underserved patients.

The biobank at CHOP’s Center for Applied Genomics (CAG) has the largest biorepositories of pediatric genetic data in the world.

Access to the genetic information in the CAG biobank will allow researchers at the two organizations 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 chief scientific officer and executive vice-president Philip Johnson said: "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.

"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."

As part for the deal, Medgenics will pay $5m to CHOP and will also 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.

In exchange, CHOP will also receive milestone payments and low single-digit royalties on future sales from products developed from the collaboration.

Medgenics will also be responsible for sponsoring further research at CHOP on rare and orphan diseases.