The cystic fibrosis player does deals with Crispr and Exonics to launch itself into a highly competitive rare disease area.
Protagonist Therapeutics and Pfizer will soon get an idea of their respective chances in beta-thalassaemia and Duchenne muscular dystrophy.
The first sickle cell and beta thalassaemia patients have been enrolled into a Crispr/Cas9 trial, heating up a race with Bluebird, Sanofi, Sangamo and Editas.
Tiny signs of a therapeutic benefit are offset by safety worries, as the company pivots to higher doses and second-generation projects.
Novartis's $4m price target for AVXS-101 fuels concerns that too many new gene therapies could overburden payers without a hard look at how they are financed.
As data on Amicus Therapeutics' clinical-stage Pompe disease candidate roll in, the group locks up preclinical gene therapy assets.
Companies that want to play in rare diseases need to shop for gene therapies, but price tags are looking rich.
Reaction to data from Sangamo's ongoing genome-editing trial shows that small numbers can equal big risks.
Clinical progress – or the lack of it – underpins smaller biotechs' Q2 stock performance.