Remove Gene Silencing Remove Protein Remove RNA
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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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Novo Nordisk snaps up RNAi partner Dicerna in $3.3bn takeover deal

pharmaphorum

Danish drugmaker Novo Nordisk must like what it has seen in its two-year-old alliance with gene-silencing specialist Dicerna Pharma – it has just agreed to acquire the biotech for $3.3 Once in the blood, the GalXC molecules travel to the liver, where they enter hepatocyte cells and can switch protein production on or off.

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Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug

pharmaphorum

The reductions matched the efficacy of current therapies for ATTR amyloidosis that require chronic dosing such as Alnylam’s Onpattro (patisiran) and Ionis/Akcea’s Tegsedi (inotersen) – both gene-silencing agents which can cost around $450,000 a year. — Eric Topol (@EricTopol) June 26, 2021.

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NICE backs Alnylam’s Amvuttra for rare form of amyloidosis

pharmaphorum

Alnylam’s gene-silencing drug Amvuttra has been recommended as a treatment for hereditary transthyretin-related (ATTR) amyloidosis by NICE, paving the way for the drug to be made available on the NHS in England and Wales.

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Epigenetic Editing with CRISPR Might Be Easier Than We Thought

XTalks

The tool could also prove to be safer than conventional CRISPR-based gene therapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. Genes can be switched on with a complementary tool called CRISPRon that has also been described in the paper. Epigenetic Editing with CRISPR.

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Unfolding The Folds Of Transthyretin

Delveinsight

Transthyretin (TTR) is a protein that the liver produces to facilitate the transfer of thyroid hormone and vitamin A in the blood. However, due to specific reasons, the TTR protein begins to misfold and aggregate as fibrils, a process known as amyloidosis of Transthyretin (ATTR). Gene-silencing Therapies.

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