Just over a month after inking a Parkinson's disease gene therapy pact with the UK's Oxford BioMedica, Axovant has licensed a 'silence-and-replace' technology from Australia's Benitec to tackle a rare but potentially fatal disease, oculopharyngeal muscular dystrophy.
Axovant is attempting to reinvent itself as a key player in gene therapy and has signed a second deal in the area in the space of a month, this time with Australian gene-silencing specialist Benitec Biopharma Ltd..
Benitec has licensed the exclusive global rights for BB-301, now named AXO-AAV-OPMD, intended for the treatment of oculopharyngeal muscular dystrophy...
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
