Patients in the EU with Fanconi anaemia (FA) could soon have the first gene therapy option for the disorder, as the EMA starts a review of Rocket Pharma’s RP-L102 candidat
Shares in Rocket Pharmaceuticals have been living up to their name, shooting up following encouraging early-stage clinical trial results from a gene therapy for a serious inherited rare hea
Neurogene has reported encouraging efficacy results with its gene therapy for rare genetic disorder Rett syndrome, but investors' concerns about safety seem to be weighing on its share pric
Investigator sites are essential for successful execution of clinical trials – ensuring studies are conducted ethically, safely, and in compliance with regulatory standards.