AstraZeneca's rare diseases unit Alexion has agreed to pay $1 billion to acquire Pfizer's early-stage gene therapy pipeline and enabling technologies - the company's first
French biotech Sensorion has filed its first regulatory application to start human testing of OTOF-GT, a gene therapy for one of the most common forms of congenital deafne
The FDA has approved BioMarin Pharma’s Roctavian as the first gene therapy for haemophilia A, giving patients an alternative to regular injections of blood clotting factor
Almost against the odds, Sarepta’s gene therapy for muscle-wasting disease Duchenne muscular dystrophy (DMD) has been given accelerated approval by the FDA, albeit in a fa
Bavarian Nordic is preparing to file for regulatory approval of a vaccine against chikungunya virus after its CHIKV VLP candidate showed protective efficacy in a second ph
To improve trial recruitment, engagement, and retention, and to increase the likelihood that trials reflect what matters most to patients, regulatory authorities increasingly point to metho