Ionis Pharmaceuticals, Inc. develops antisense therapies, RNAi, and CRISPR therapeutics from Carlsbad, California; known since 1989 as Isis Pharmaceuticals until 2015.
AstraZeneca has announced that Wainua, its gene-silencer drug for transthyretin-mediated amyloid cardiomyopathy, did not meet its primary endpoint in a phase 3 trial. The news triggers a sharp stock drop and prompts investors to rethink the company’s long-term growth targets and the strength of its pipeline.
A body of studies is showing that a blood marker called p-tau217 can forecast an elevated risk of cognitive impairment in healthy older adults years before symptoms appear. The evidence is not yet clinical-ready, but it is guiding trials that aim to slow or prevent Alzheimer’s disease and is being tested in Scotland’s primary care setting.