AT132 has prompted more safety concerns, this time at a lower dose than before.
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.