Gene and cellular therapies that can replace decades of expensive therapy do not come cheap. Even the initial remedy of payment by installments could be irrelevant when not all patients respond in the same way.

No new patients will be enrolled, and trial participants will stop treatment with the tissue factor pathway inhibitor antibody, a class with four prior suspended hemophilia programs.

After last week's headline-grabbing deal between Bristol Myers Squibb (BMS) and Dutch gene therapy company uniQure (scripintelligence.com, 6 April 2015), who would have thought that events in Europe might by the end of the week spell the demise of uniQure's gene therapy platform?

Non-profit hospital spins out its first start-up, which will house one program to address a heritable form of blindness, another to treat hemophilia B.