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Novo Nordisk bulks up in rare diseases with $1.2bn Prothena deal

pharmaphorum

The Danish drugmaker is paying $100 million upfront for rights to antibody drug PRX004, which is heading for phase 2 trials in cardiomyopathy associated with ATTR amyloidosis, a life-threatening disease affecting the heart and nervous system.

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Novartis’ big bet on inclisiran nears fruition, as CHMP backs drug

pharmaphorum

If approved, inclisiran would become the first and only gene-silencing drug to reduce low-density lipoprotein cholesterol (LDL-C) in these patients. The drug inhibits PCSK9 – the same target as Amgen’s Repatha (evolocumab) and Sanofi/Regeneron’s Repatha (alirocumab) – but is dosed only twice a year rather than every month.

Drugs 98
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Cash round sets up trial of InteRNA’s microRNA for cancer

pharmaphorum

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.

Trials 59
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Novartis’ big bet on inclisiran nears fruition, as CHMP backs drug

pharmaphorum

If approved, inclisiran would become the first and only gene-silencing drug to reduce low-density lipoprotein cholesterol (LDL-C) in these patients. The drug inhibits PCSK9 – the same target as Amgen’s Repatha (evolocumab) and Sanofi/Regeneron’s Repatha (alirocumab) – but is dosed only twice a year rather than every month.

Drugs 52
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Unfolding The Folds Of Transthyretin

Delveinsight

Gene-silencing Therapies. Silencing or modifying gene TTR aims to reduce the effect of both variant and wild-type TTR gene, thus reducing hepatic production by targeting its mRNA. The past years have witnessed a shift from off-label and symptomatic therapies to approved TTR stabilizers and gene-silencing therapies.

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NICE backs NHS use of Novartis’ cholesterol drug Leqvio

pharmaphorum

In its guidance, NICE said there is enough evidence to support Leqvio in people with ASCVD, although there is no data directly comparing the drug with other treatments like Organon’s Ezetrol (ezetimibe) and PCSK9 antibodies Praluent (alirocumab) from Sanofi/Regeneron and Amgen’s Repatha (evolocumab).

Drugs 98
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2020 review – Pharma’s progress outside of COVID-19

pharmaphorum

In summer, Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder Oxlumo was approved in the European Union and the US. The antibodies produced as a result lodge in the kidneys, causing severe complications. Rare disease progress.