Trace Neuroscience, which launched this week with $101 million, benefited from last year’s approval of Qalsody. Its CEO spoke ...

The FDA has accepted for review the resubmitted BLA for prademagene zamikeracel for recessive dystrophic epidermolysis bullosa.

Put up against placebo in the phase 3 EMBARK trial, delandistrogene moxeparvovec (Elevidys) did not significantly improve ...

Why one trial participant's healthy birth after her CAR-T treatment for lupus is catching resesarchers' attention: ...

Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...