Promising early data with Sangamo and Pfizer’s haemophilia A gene therapy could set up a battle with Roche and Biomarin.
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…
Promising data with Fibrocell’s epidermolysis bullosa gene therapy send the company down as funding worries kick in.
With rich countries like the US finding it hard to fund gene therapies, it is worth asking whether these projects will ever reach patients in developing countries. And if…
Three gene therapy contenders, Abeona, Fibrocell and Krystal, are taking different approaches in the quest to cure epidermolysis bullosa.
Axovant is the latest company to benefit from excitement over gene therapy data in small numbers of patients.
Krystal Biotech shares surged after positive data with its epidermolysis bullosa project, KB103, in just two patients. Now it needs to repeat the trick.
Biogen’s $877m buyout of Nightstar Therapeutics provides yet more evidence that gene therapy is reaching critical mass.
Keenly awaited results from the first trial of MYO-101 broadly beat expectations, leading Sarepta to buy the gene therapy’s originator.