Protagonist Therapeutics and Pfizer will soon get an idea of their respective chances in beta-thalassaemia and Duchenne muscular dystrophy.
A run of bad news leaves investors questioning Wave Life Sciences’ ability to deliver on its promises.
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.
Tiny signs of a therapeutic benefit are offset by safety worries, as the company pivots to higher doses and second-generation projects.
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.
Bluebird Bio has pushed forward the conversation on how to make million-dollar treatments affordable for insurers, but biopharma and payers are ominously quiet about…