Keyword: Spark Therapeutics
Spark Therapeutics announced encouraging preclinical data on SPK-3006, its gene therapy to treat the muscle disorder Pompe.
ProQR Therapeutics reported interim phase 1/2 data for its LCA10 treatment showing it improved vision for the majority of patients.
uniQure has treated the first of around three patients due to enroll in its phase 2b dose-confirmation trial of hemophilia B gene therapy AMT-061.
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.