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.
What was up is now down as smaller biotech stocks deal with clinical trial failure and acquisitions.
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…
Keenly awaited results from the first trial of MYO-101 broadly beat expectations, leading Sarepta to buy the gene therapy’s originator.
In the race to develop a haemophilia B gene therapy Uniqure now has the upper hand.
Solid Biosciences’ muscular dystrophy gene therapy fails to hit the company’s own target, let alone a benchmark set by Sarepta.
A limb-girdle muscular dystrophy gene therapy Sarepta licensed last year faces its first clinical test.
Solid Biosciences should soon find out if its DMD gene therapy, SGT-001, could compete with Sarepta’s.