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.
Topsy turvy times for industry’s mid and small cap stocks in the first quarter
What was up is now down as smaller biotech stocks deal with clinical trial failure and acquisitions.
Should gene therapy patients be asked to pay?
The price in value-based payment deals should by definition be justified, so why is patient cost-sharing part of the calculation? Spark covers this cost, but there is no…
Myonexus becomes the third string to Sarepta’s muscular dystrophy bow
Keenly awaited results from the first trial of MYO-101 broadly beat expectations, leading Sarepta to buy the gene therapy’s originator.
Uniqure turns the screw on Spark and Pfizer
In the race to develop a haemophilia B gene therapy Uniqure now has the upper hand.
Gene therapy results look less than Solid
Solid Biosciences’ muscular dystrophy gene therapy fails to hit the company’s own target, let alone a benchmark set by Sarepta.
Moment of truth for Sarepta and Myonexus
A limb-girdle muscular dystrophy gene therapy Sarepta licensed last year faces its first clinical test.
Solid guns for Sarepta
Solid Biosciences should soon find out if its DMD gene therapy, SGT-001, could compete with Sarepta’s.
Kickstarting ideas
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