Phase 1
Shares of Incyte Corporation are up slightly this morning after the company started off its annual R&D day with positive news from its pivotal REACH1 study of Jakafi (ruxolitinib) in steroid-refractory acute graft-versus-host disease (GVHD) and sets up the filing of a supplemental New Drug Application.
Sarepta Therapeutics shares rocketed by as much as 80 percent yesterday after the company released interim data on its experimental gene therapy AAVrh74.MHCK7.micro-Dystrophin for Duchenne muscular dystrophy (DMD).
Chinese companies and investors continue to flex their financial muscles with a series of deals this week that highlight the continued growing impact that country is having on the global pharmaceutical and biotech landscape.
PTC Therapeutics presented updated interim clinical data from Part 1 of its FIREFISH clinical trial of risdiplam (RG7916) in babies with Type 1 Spinal Muscular Atrophy.
The U.S. Food and Drug Administration (FDA) placed a clinical hold on Boston-based Ziopharm Oncology’s Phase I trial of its Sleeping Beauty CAR-T therapy.
Shares of Solid Biosciences have jumped 11 percent in premarket trading after the company announced the U.S. Food and Drug Administration has lifted a clinical hold on the company’s Phase I/II clinical trial gene therapy treatment for Duchenne muscular dystrophy (DMD).
Bluebird bio’s LentiGlobin gene therapy is increasing hemoglobin levels in anemia patients battling sickle cell disease and beta thalassemia, which reduces the need for blood transfusions in those patients.
While pharma and biotech researchers have been driving forward with therapies aimed at helping Parkinson’s disease patients, medical researchers have identified “unexpected new key players” in the development of early-onset Parkinson’s disease, called Parkinsonism.
Alexion Pharmaceuticals is forging a partnership with Complement Pharma to co-develop a C6 complement inhibitor.
Unity Biotechnology has raised $222 million in venture capital, raised $85 million in an initial public offering (IPO) in May, and has a value of $700 million. All without having a product to sell.
PRESS RELEASES