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.
Wave crashes again – this time on toxicity concerns
A run of bad news leaves investors questioning Wave Life Sciences’ ability to deliver on its promises.
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.
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.
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.
Gene therapy: how much will it cost patients?
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…
Kickstarting ideas
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