bluebird bio started last week on tenterhooks, as FDA advisors cast their eyes over data for two gene therapy candidates that are key to its future as a commercially viable concern.
The FDA may have safety concerns abut bluebird bio's gene therapy for rare, fatal disease cerebral adrenoleukodystrophy (CALD), but its advisors believe its benefits far outweigh the risks.
PTC Therapeutic's Upstaza gene therapy for patients with genetic disorder AADC deficiency has been recommended for approval in the EU, setting up another test of the commercial prospects f
SwanBio Therapeutics has raised $56 million in a second-round financing that will be used to advance its lead gene therapy candidate for adrenomyeloneuropathy (AMN) into clinical testing la
Astellas has said it will book a $170 million impairment charge in its fourth quarter results as a result of a decision to halt the development of three gene therapy candidates for Duchenne