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Two years after starting to work together, Novo Nordisk and Dicerna have selected the first candidate from a joint project to find new, gene-silencing drugs for liver-related cardiometabolic diseases. In 2019 the Danish company also licensed a preclinical-stage, small-molecule candidate for NASH from Japan’s UBE.
The pharma group will provide funding for the research in return for exclusive license options on the programmes. billion takeover of gene-silencing specialist Dicerna Pharma last year, another deal aimed at extending its R&D capabilities by bolting on a new platform technology – in this case RNA interference (RNAi) drugs.
InteRNA is focusing on the development of gene-silencing drugs based on microRNA, naturally occurring, non-coding strands of RNA that are thought to regulate gene expression in cells. Regulus meanwhile remains a microRNA player, but has suffered a number of setbacks.
The US biotech has just reported phase 2 trial results with the small, interfering RNA (siRNA) gene-silencing drug showing that it an cause a 90% or greater reduction in Lp(a) levels – a risk factor for cardiovascular disease – that was sustained over 48 weeks of follow-up.
The FDA has approved Alnylam’s genesilencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder. Alnylam licensed inclisiran to The Medicines Company, which was bought last year by Novartis for $9.7
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