pharmaphorum

JULY 13, 2021

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. The post Novo Nordisk bulks up in rare diseases with $1.2bn Prothena deal appeared first on.

Gene Silencing 98

Gene Silencing Sales Gene Editing Clinical Trials 98

pharmaphorum

FEBRUARY 9, 2021

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.

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Trials Gene Silencing RNA Gene Expression 58

pharmaphorum

OCTOBER 19, 2020

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 93

Drugs Gene Silencing Sales Antibody 93

pharmaphorum

OCTOBER 19, 2020

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

Drugs Gene Silencing Sales Antibody 52

Delveinsight

AUGUST 6, 2020

Hereditary ATTR amyloidosis , also known as Familial ATTR, is an autosomal dominant disease and a result of mutations in the TTR gene, thus it is passed down from one generation to the next one. Gene-silencing Therapies. ATTR amyloidosis can be either hereditary or acquired (non-hereditary). hATTR market : In The Pipeline.

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Gene Silencing Protein Marketing Gene 52

pharmaphorum

DECEMBER 23, 2020

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.”. The antibodies produced as a result lodge in the kidneys, causing severe complications.

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Marketing Gene Silencing Gene Drugs 75

pharmaphorum

SEPTEMBER 1, 2021

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 90

Drugs Trials Gene Silencing Genetics 90