Vertex’s second transformation: Duchenne time
The cystic fibrosis player does deals with Crispr and Exonics to launch itself into a highly competitive rare disease area.
Upcoming events – Protagonist and Pfizer target rare diseases
Protagonist Therapeutics and Pfizer will soon get an idea of their respective chances in beta-thalassaemia and Duchenne muscular dystrophy.
Vertex and Crispr up the ante in gene therapy chase
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.
Higher dose points Sangamo no closer to zinc finger success
Tiny signs of a therapeutic benefit are offset by safety worries, as the company pivots to higher doses and second-generation projects.
How many million-dollar drugs can health systems tolerate?
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.
Amicus muscles in on Pompe gene therapy chase
As data on Amicus Therapeutics' clinical-stage Pompe disease candidate roll in, the group locks up preclinical gene therapy assets.
Gene therapy deal-making shows no signs of stopping
Companies that want to play in rare diseases need to shop for gene therapies, but price tags are looking rich.
Zinc fingers get the thumbs down
Reaction to data from Sangamo's ongoing genome-editing trial shows that small numbers can equal big risks.
Mid and small caps provide another rocky ride in the second quarter
Clinical progress – or the lack of it – underpins smaller biotechs' Q2 stock performance.
Kickstarting ideas
Independent, data-driven daily news and analysis on pharma, biotech and medtech.