Better-looking safety might not be enough to get Solid back in the Duchenne muscular dystrophy game.
Sarepta’s first ever controlled study, of a Duchenne muscular dystrophy gene therapy, brings the markets down to earth.
Drug makers from across the globe feature in 2019’s list of best performing small biotech stocks, from Galapagos to Daiichi Sankyo to Arrowhead.
An analysis of company disclosures reveals a sharp rise in US FDA-imposed halts on clinical work involving gene therapies.
Wave will soon report key early-stage data with its exon-skipping DMD candidate suvodirsen but, unlike Sarepta, the group is starting its confirmatory trial before…