Novartis reported that its intrathecal (IT) formulation of onasemnogene abeparvovec (OAV101 IT) reached its primary endpoint in a Phase III study in paediatric patients aged two to 17 with type II ...
Astellas' aspirations in gene therapy have been knocked back by a series of setbacks, including cases of liver toxicity and deaths in a trial of its AT132 gene therapy candidate for rare disease X ...