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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 98
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Unfolding The Folds Of Transthyretin

Delveinsight

The Hereditary Transthyretin Amyloidosis (hATTR) Market size in the 7MM was estimated to be USD 306.9 Among the 7MM geographies, the US accounted for the 54% share of the total hATTR market size. hATTR Market: Present Treatment Options. ATTR amyloidosis can be either hereditary or acquired (non-hereditary). TTR Stabilizers.

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

pharmaphorum

2020 also saw some of the first “tumour agnostic” cancer drugs get to market, with Bayer’s Vitravki (larotrectinib) getting funding in the UK for tumours with confirmed neurotrophic tyrosine receptor kinase (NTRK) gene fusions.”.

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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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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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RNA Therapeutics: A Novel Approach to Treating Diseases

Roots Analysis

Coding RNAs include messenger RNA (mRNA) and short interfering RNA (siRNA), which encode proteins and silence gene expression, respectively. RNA interference (RNAi) Therapeutics : Fundamentally, RNAi is a natural process of post-transcriptional gene silencing, involving short strands of nucleic acids.

RNA 40