Two haemophilia A subjects help Sangamo erase 2019's losses
Promising early data with Sangamo and Pfizer’s haemophilia A gene therapy could set up a battle with Roche and Biomarin.
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…
Fibrocell gets a reality check
Promising data with Fibrocell’s epidermolysis bullosa gene therapy send the company down as funding worries kick in.
How to pay for gene therapies in developing nations
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…
Gene therapies go skin deep to tackle epidermolysis bullosa
Three gene therapy contenders, Abeona, Fibrocell and Krystal, are taking different approaches in the quest to cure epidermolysis bullosa.
Axovant reaps the rewards from its gene therapy refocus
Axovant is the latest company to benefit from excitement over gene therapy data in small numbers of patients.
Krystal gets more skin in the epidermolysis bullosa game
Krystal Biotech shares surged after positive data with its epidermolysis bullosa project, KB103, in just two patients. Now it needs to repeat the trick.
Nightstar gives Biogen a cheap way to expand in gene therapy
Biogen’s $877m buyout of Nightstar Therapeutics provides yet more evidence that gene therapy is reaching critical mass.
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.
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