After recent landmark approvals in other neurodegenerative diseases such as Alzheimer’s and amyotrophic lateral sclerosis, hopes are rising that Huntington’s disease could be next to reach a breakthrough.
UniQure is awaiting updated data from its potential first-in-class gene therapy, AMT-130, with the most important being a Phase I/II trial in the US monitoring a small group of patients at 12 and 24 months after treatment.
The gene therapy uses an AAV5 vector to transports a micro-RNA into the central nervous system with the goal of...
Biogen hopes to move directly from Phase I to Phase III with salanersen, a higher-potency therapy that uses the same mechanism of action as Spinraza.
Oral RNA splicing modulator has gone into a Phase II/III trial
Wales-based group launches with $140m series A.
Supernus agreed to pay $561m upfront plus a contingent value right that could add $234m to buy Sage, topping a previously rejected offer from Biogen.
The late-stage biotech posted results from the maintenance extension portion of its Phase IIb QUALITY trial in which enobosarm reduced weight regain and helped preserve lean mass in patients who discontinued their semaglutide weight-loss treatment.
GSK tells Scrip about timelines for data expected from two Phase II trials for its Bharat Biotech-partnered shigella vaccine candidate, which puts it ahead of Limmatech/Valneva. Meanwhile, a global-first malaria vaccine, also partnered with Bharat, sees an over 50% price cut
The drugmaker announced positive topline results for the drug in hemophilia A and B patients with inhibitors, on top of the existing approval for patients without inhibitors.
