Biomarin suffers minimal bleeding on valrox readout
Three-year data from Biomarin’s haemophilia A gene therapy fail to quash concerns about durability, but the company insists that the results are good enough to file on.
Biomarin hopes long-term valrox data can stem the bleeding
Expectations are low for a three-year update on the group’s haemophilia A gene therapy, and Biomarin’s rivals could capitalise on any slip-up.
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…
Roche buy-in is another vote of confidence in gene therapy
The Swiss group picks up Spark Therapeutics for $4.8bn, marking the biggest gene therapy buyout since Novartis’s $8.7bn move on Avexis.
Last year’s new medicines reveal strong commercial promise and some novelty
The commercial potential of last year’s new medicines suggests another strong year of productivity from biopharma, while a further analysis reveals the arrival of two…
Tiny populations up the ante for gene therapy pricing
Digging into the sales forecasts for gene therapies shows just how low their penetration is expected to be. Could biopharma come to regret its high-price, low-volume…
Ash 2018 preview – Freeline ziplines into haemophilia gene therapy
The private UK group could steal the thunder of rivals like Spark, Uniqure and Biomarin.
How many million-dollar drugs can health systems tolerate?
Novartis's $4m price target for AVXS-101 fuels concerns that too many new gene therapies could overburden payers without a hard look at how they are financed.
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