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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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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Scienmag

JANUARY 4, 2021

The findings could yield important implications for development of drugs to treat cancer and other diseases. 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).

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pharmaphorum

JUNE 28, 2021

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . — Eric Topol (@EricTopol) June 26, 2021. In three more patients treated at 0.1mg/kg levels fell by 52%.

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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 Dicerna has been a hot tip for takeover for a couple of years, as the list of approved drugs that work via RNAi has lengthened. billion in cash.

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pharmaphorum

JANUARY 19, 2023

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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pharmaphorum

JULY 13, 2021

Diabetes giant Novo Nordisk has opened a new front in its rare disease R&D, thanks to an acquisition agreement with Prothena focusing on drugs for ATTR amyloidosis. In ATTR amyloidosis, transthyretin (TTR) protein misfolds and accumulates as amyloid deposits throughout the body. billion by 2025.

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