Shares in Solid Biosciences plunged yesterday after the FDA placed a clinical hold on the trial of its gene therapy for Duchenne muscular dystrophy (DMD) following a serious adverse reactions in one patient.
Jay Bradner, head of Novartis’ drug discovery, talks to pharmaphorum about the firm’s drive to transform itself into 'a medicine and data science company'.
The trial of Sangamo’s gene-editing treatment for a rare and debilitating disease has got off to a good start, with no safety concerns reported so far.
Spark Therapeutics is on the cusp of gaining the first ever approval for a gene therapy from the FDA, a groundbreaking treatment which restores vision to children and adults with a rare inh
A pharma company’s core mission is to improve patient outcomes. This hinges on effectively influencing HCP clinical behaviour and driving disease education.
