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Novo Nordisk’s gene silencing alliance with Dicerna bears first fruit

pharmaphorum

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. The post Novo Nordisk’s gene silencing alliance with Dicerna bears first fruit appeared first on.

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RNA Therapeutics: A Novel Approach to Treating Diseases

Roots Analysis

RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. Non-coding RNAs include antisense oligonucleotides (ASOs) and RNA aptamers.

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FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1

The Pharma Data

Oxlumo works by degrading HAO1 messenger RNA and reducing the synthesis of GO, which inhibits hepatic production of oxalate – the toxic metabolite responsible for the clinical manifestations of PH1. HAO1 encodes glycolate oxidase (GO), an enzyme upstream of the disease-causing defect in PH1.