Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania have announced a major expansion to their R&D collaboration for gene therapies.
BioMarin is set to file for approval for its haemophilia gene therapy valoctocogene roxaparvovec, after it achieved pre-specified clinical criteria for regulatory review.
Massachusetts holding company ElevateBio has launched with $150m in initial funding from a range of investors on a mission to build a portfolio of cell and gene therapy companies.<
A second death in a study of Novartis’ spinal muscular atrophy gene therapy Zolgensma (onasemnogene abeparvovec-xioi; AVXS-101) – a potential blockbuster for this rare disease – has prompte