In an effort to better understand diseases that have long eluded diagnosis, the New York Stem Cell Foundation Research Institute is partnering with the National Institutes of Health's Undiagnosed Diseases Program to create cell models of rare medical conditions.
Shire is one of those companies that's always rumored to be a buyer or a seller. Not so long ago, it was setting up poison-pill protections against an unwanted suitor. Now, it has beat out Big Pharma with a deal for ViroPharma, the maker of rare disease drugs.
The buyout--at $50 a share--will add Cinryze for hereditary angioedema to Shire's product lineup along with the experimental drug maribavir for cytomegalovirus, one of 8 clinical-stage programs in the pipeline.
While bioinformatics lies at the leading edge of biological research, it is still underpinned by one of the oldest tools available to humanity: communication. Recognizing this, researchers are trying to standardize terminology through the creation of a rare genetic disease dictionary.
Fidelity Biosciences is placing a big bet on gene therapy, funding the launch of startup Dimension Therapeutics with some licensed patents and a plan to develop novel treatments for hemophilia.
So is Roche going to do some rare disease deals or not? Rumors of a possible buyout of Alexion and BioMarin--alternatively dissed and touted--helped make this one of the big questions for deal watchers. Roche CEO Severin Schwan's answer can now be summarized in one word: Maybe.
Eli Lilly has found a new path into the hot rare-disease field. An NIH program has backed the Indianapolis-based drug giant's preclinical-stage research of a potential treatment for a hormone deficiency often caused by a genetic defect.
In the ongoing hunt to uncover new therapies for rare diseases, the NIH has selected four new preclinical drug development studies to support, the findings of which will be shared with other researchers.
Novartis has picked up its option on a Phase II-ready therapy for a pair of rare disease from Sweden's Camarus, forking over a milestone included in their $700 million pact inked two years ago.
Neurocrine shares took a nasty drubbing after the biotech announced that its low-dose version of an experimental therapy for a rare movement disorder failed a Phase IIb study.