Ulefnersen slows ALS decline in pivotal FUS gene trial
A Phase 3 trial finds ulefnersen, an RNA-targeted therapy from Otsuka and Ionis Pharmaceuticals, significantly slows disease progression in patients with FUS-associated ALS.
Ionis Pharmaceuticals 1 article
Within nucleic-acid medicine, Ionis Pharmaceuticals operates as an RNA-therapeutics biotechnology company focused on RNA-targeted therapeutics and supported by antisense-mediated RNA modulation. The platform also incorporates RNase H antisense mechanism and ligand-conjugated antisense technology, with intrathecal oligonucleotide delivery defining an important chemistry, delivery or RNA-processing feature of the therapeutic approach. RNA interference, antisense and other transcript-level methods remain distinct tags; the RNA platform of Ionis Pharmaceuticals also includes cardiovascular antisense therapeutics.