Michigan State University (MSU) College of Medicine investigator Bin Chen just was awarded $2.1 million by the National Institutes of Health (NIH) to scour vast databases of existing drugs in a quest to treat orphan diseases. The investigative team received an...
Orphan disease
Category
FDA Clears IND for ABO-202 Gene Therapy Candidate for Infantile Batten Disease
The FDA’s positive decision clears Abeona Therapeutics’ Investigatory New Drug Application (IND) to start a Phase I/2 trial. The studies will be conducted at infantile Batten disease global centers of excellence which include: University of Rochester Medical Center ...
Pfizer to Buy Rare Disease Portfolio Pipeline for $810 Million
Pfizer will buy Therachon Holding, a privately held, clinical-stage biotechnology company focused on rare diseases, with assets in development for the treatment of achondroplasia and short bowel syndrome (SBS). The deal is worth up to $810 million. Key Terms $340...