Novartis’ bid to expand the company’s $2.1 million-per-patient gene therapy Zolgensma to more spinal muscular atrophy patients faces a possible delay after U.S. regulators requested another study in older children getting the drug via a spinal infusion.
The adverse events that led to a pause in trials evaluating AstraZeneca Plc’s Covid-19 vaccine candidate may not have been associated with the vaccine itself, according to a document outlining participant information that was posted online by the Oxford University.
San Francisco-based Unity Biotechnology launched a restructuring program to focus on cellular senescence in ophthalmology and neurology.
AstraZeneca resumed British clinical trials of the company’s Covid-19 vaccine, one of the most advanced in development, after getting the green light from safety watchdogs.
bluebird bio Inc. announced updated results from the clinical development program for the company’s investigational elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy in patients with cerebral adrenoleukodystrophy (CALD), including long-term results from the Phase 2/3 Starbeam study and data from the Phase 3 ALD-104 study.
Shares of Reata Pharmaceuticals plunged more than 33 percent after the company announced a potential delay in plans to seek approval of omaveloxolone, an investigational treatment of Friedreich’s ataxia (FA).
Biogen successfully fended off a patent challenge from generic company Mylan over the multiple sclerosis (MS) drug Tecfidera.
Reata Pharmaceuticals Inc.’s Part 2 portion of the MOXIe Phase 2 trial of omaveloxolone in patients with Friedreich’s ataxia (FA) met the primary endpoint of change in the modified Friedreich’s Ataxia Rating Scale relative to placebo after 48 weeks of treatment.
UCB will acquire U.S.-based Ra Pharmaceuticals in a $2.1 billion deal that will enable the Belgian drugmaker to offer new treatment options for several rare diseases in neurology and immunology.
German drugmaker Bayer is paying up to $600 million for full control of cell therapy developer BlueRock Therapeutics.