Keyword: Spark Therapeutics
Sigilon Therapeutics is bringing on Bioverativ’s chief global therapeutic operations officer, Rogerio Vivaldi, M.D., to serve as its CEO.
Spark Therapeutics’ hemophilia A gene therapy reduced bleeding by 97% in a phase 1/2 trial but sent one patient to the hospital.
Early data suggest the new batch of therapies is at least as effective as its predecessor, clearing a potential pitfall on Spark’s path to market.
A Salk Institute team found that transplanting liver cells into hemophilic mice restored their ability to form blood clots.
Spark Therapeutics agreed to sell its priority review voucher, good for four months off an FDA review, to Jazz Pharmaceuticals for $110 million.
The round sets up the JLABS resident to use advances in synthetic biology to create more targeted, controllable cell and gene therapies.
Just over a week ago, Spark Therapeutics was riding high on positive data for its hemophilia B gene therapy. It’s now fallen back to earth after spooking investors with the results of a trial in hemophilia A.
Patients with hemophilia B have seen their symptoms controlled for up to a year with a single dose of a gene therapy developed by Spark Therapeutics, almost entirely eliminating the need for treatment with clotting factors.
The mid-development tweaks have the blessing of regulators, enabling uniQure to start a pivotal trial next year with its fast-track statuses intact.
After decades of research, a gene therapy to correct a specific genetic mutation is on the brink of being approved by the FDA.