Vertex spends nearly $1bn on Semma's unproven diabetes cell tech
Having succeeded with cystic fibrosis Vertex is now chasing a cure for type 1 diabetes.
EHA 2019 – Global Blood faces sickle cell questions
More data with voxelotor shouldn’t hurt the project’s bid for accelerated approval, but there are still doubts about its effect on a harder patient outcome.
Emmaus tries to move forwards by reversing
The company insists that its sickle cell disease therapy Endari still has a future, despite questionable efficacy and the approach of new treatments.
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.
Kickstarting ideas
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