Pharmaceutical Technology

FEBRUARY 20, 2023

The therapy is specifically aimed at treating FTD patients who have mutations in their granulin gene, which encodes for the protein progranulin (PGRN). This protein promotes lysosomal function. In addition to gene therapy, gene silencing, which can be done using ASOs, is also an interesting approach, says Barmada.

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pharmaphorum

JANUARY 6, 2021

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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Gene Silencing Gene RNA Contract Manufacturing 60

Scienmag

JANUARY 4, 2021

BOSTON – A long-running debate over how an important gene-silencing protein identifies its targets has been resolved by researchers at Massachusetts General Hospital (MGH). The findings could yield important implications for development of drugs to treat cancer and other diseases.

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pharmaphorum

JUNE 28, 2021

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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In-Vivo Gene Editing Trials Drugs 101

pharmaphorum

JULY 13, 2021

In ATTR amyloidosis, transthyretin (TTR) protein misfolds and accumulates as amyloid deposits throughout the body. In 2019, for example it agreed to work with Dicerna on gene-silencing for liver-related cardio-metabolic disease in a deal that involved a $225 million upfront payment.

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XTalks

MAY 4, 2021

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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pharmaphorum

NOVEMBER 18, 2021

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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