Data will soon show whether Avadel’s FT218 is more convenient than Jazz’s Xyrem, while Axovant will be looking for a dose response with its Parkinson’s gene therapy.
Despite the recent flurry of gene therapy deals there is still a fairly extensive list of affordable companies out there.
The usual story of positive data and clinical failure influencing company stock performance was given added interest in the second quarter with price fixing allegations…
The Swiss group picks up Spark Therapeutics for $4.8bn, marking the biggest gene therapy buyout since Novartis’s $8.7bn move on Avexis.
A collaboration over four gene therapy programmes substantially beefs up Neurocrine’s pipeline, while Voyager Therapeutics gets a much-needed infusion of credibility.
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.
Companies that want to play in rare diseases need to shop for gene therapies, but price tags are looking rich.