We presented progress on our RNAi therapeutics platform efforts at the Oligonucleotide Therapeutics Society (OTS) 2021 Annual Meeting. Read the press release Harnessing RNAi for a New Class of Medicines – John Maraganore Expanding the Reach of RNAi Therapeutics – Kirk Brown Development of Lumasiran for the Treatment of...
New kidney stone data from ongoing clinical trials of OXLUMO® (lumasiran), an RNAi therapeutic targeting hydroxyacid oxidase 1 – the gene encoding glycolate oxidase – for the treatment of primary hyperoxaluria type 1 (PH1), were presented at the American Urological Association (AUA) Annual Meeting, held...
Additional positive 9-month results from subgroup analyses and exploratory endpoints of the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis with polyneuropathy, were presented at the 3rd EU-ATTR Amyloidosis Meeting. Subgroup analyses and exploratory...
Positive results from a Phase 3b open-label study of patisiran in hereditary ATTR amyloidosis patients with polyneuropathy progression after receiving an orthotopic liver transplant were presented at the Peripheral Nerve Society’s 2021 Annual Meeting. Read our press release View the presentation...
