Avrobio dropping out of the race means Sangamo can take advantage but its gene therapy must show long-term efficacy in patients not receiving enzyme replacement therapy.
After its biggest competitor AVROBIO fell behind in the battle to develop the first ever Fabry disease gene therapy, Sangamo Therapeutics could now be in a position to win the race to market.
The Brisbane, CA-based company has provided an update on a Phase I/II trial of its candidate ST-920 (isaralgagene civaparvovec), with...
A court order encompassing funding, drug pricing, clinical trials and overall policy implementation aspects is expected to bring about a paradigm shift in the treatment of rare diseases in India. Sarepta, Roche and Sanofi are among the key players that have been part of pricing discussions.
Merck & Co. won European Commission approval for Winrevair on top of other medicines for pulmonary arterial hypertension and expects an initial EU launch in Germany.
The company, in its new profitable era, is bringing in new leadership, with Amgen VP of medical affairs for rare diseases Greg Friberg and recently retired Roche Partnering head James Sabry.
Bluebird, Vertex and Sarepta are starting to see some commercial gene therapy traction and expect momentum to pick up in the second half, while hemophilia remains a challenging area.
Lilly's first Phase III triplet win opens the door to a label expansion in BTK-pretreated CLL, with analysts suggesting Jaypica could attain blockbuster status. However, the window to capitalize may be brief as next-generation BTK degraders close in with head-to-head trials.
Mounjaro, Eliquis, and Darzalex buck the Q1 trend to make gains
