News & Analysis on the Clinical Development and Manufacture of Large Molecule Drugs
Sarepta Therapeutics has hit a setback as its approved gene therapy Elevidys missed the primary goal of a phase 3 trial in children aged 4 through 7 years with the inherited disease Duchenne muscular dystrophy (DMD).
The French biotech company Vect-Horus has licensed its technology to the Danish big pharma Novo Nordisk to deliver drug cargoes to undisclosed disease targets in three programs.
The London and New York-based gene therapy player MeiraGTx Holdings has received a $30 million investment from Sanofi in addition to potential strategic deals with the big pharma company down the road.