Keyword: Spark Therapeutics
The update suggests FIX activity increases seen at six weeks are sustainable, giving uniQure a boost as it races Spark Therapeutics to market.
After refocusing on gene therapy, Axovant is appointing five new executives to posts ranging from clinical development to commercial operations.
The FIX-Padua therapy achieved FIX activity levels of 23% to 37%, suggesting it is several times more effective than unIQure's old asset.
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.