Skip to main content
Premium Trial:

Request an Annual Quote

Medgenics, CHOP Collaborate on Pediatric Rare, Orphan Genetic Diseases

NEW YORK (GenomeWeb) – Medgenics announced on Wednesday a collaboration with the Children's Hospital of Philadelphia to accelerate the development of new therapies for pediatric rare and orphan genetic diseases.

Under the agreement, Medgenics will have access to the biobank at CHOP's Center for Applied Genomics (CAG), one of the world's largest biorepositories of pediatric genetic data, which will enable researchers at both organizations to identify new rare and orphan disease targets and accelerate the development of new therapies into clinical stage programs.

Medgenics will pay CHOP $5 million in exchange for an exclusive license to use the rare and orphan disease samples at the CAG biobank to develop and commercialize therapeutic treatments and diagnostic targets. CHOP will also receive milestone payments and low single-digit royalties on future sales from products developed from the agreement. Medgenics will sponsor further research at CHOP on rare and orphan disease.

CHOP CEO Steven Altschuler noted that about 10 percent of all rare and orphan disease patients in North America go through his facility, which will provide CHOP and Medgenics "opportunities to link diseases and their genetic abnormalities with potential new treatment paradigms," he said in a statement.

Philip Johnson, CSO and executive vice president at CHOP, added 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 toward treating the underlying causes of these diseases."

The Scan

LINE-1 Linked to Premature Aging Conditions

Researchers report in Science Translational Medicine that the accumulation of LINE-1 RNA contributes to premature aging conditions and that symptoms can be improved by targeting them.

Team Presents Cattle Genotype-Tissue Expression Atlas

Using RNA sequences representing thousands of cattle samples, researchers looked at relationships between cattle genotype and tissue expression in Nature Genetics.

Researchers Map Recombination in Khoe-San Population

With whole-genome sequences for dozens of individuals from the Nama population, researchers saw in Genome Biology fine-scale recombination patterns that clustered outside of other populations.

Myotonic Dystrophy Repeat Detected in Family Genome Sequencing Analysis

While sequencing individuals from a multi-generation family, researchers identified a myotonic dystrophy type 2-related short tandem repeat in the European Journal of Human Genetics.