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 98

Drugs Gene Silencing Sales Antibody 98

pharmaphorum

OCTOBER 29, 2020

Already a major player in gene therapy, Novartis has swooped on US startup Vedere Bio in a $280 million deal that builds its position in inherited eye diseases that can lead to blindness. Novartis’ big move in the gene therapy market came when it bought AveXis for $8.7 It also gets two preclinical-stage development projects.

Gene Therapy 40

Gene Therapy Gene Gene Silencing Protein 40

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.”.

Marketing 97

Marketing Gene Gene Silencing Drugs 97

pharmaphorum

JUNE 1, 2022

The US biotech has just reported phase 2 trial results with the small, interfering RNA (siRNA) gene-silencing drug showing that it an cause a 90% or greater reduction in Lp(a) levels – a risk factor for cardiovascular disease – that was sustained over 48 weeks of follow-up.

Gene Silencing 59

Gene Silencing Drugs Medicine RNA 59

Pharma Marketing Network

DECEMBER 21, 2020

those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies. This relinquishes many of the perceived benefits of RNAi therapies that distinguish them from gene therapy (e.g.,

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

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.

Trials 59

Trials Gene Silencing RNA Gene Expression 59

pharmaphorum

APRIL 13, 2021

There are several competitors developing treatments for hepatitis B in what could turn out to be a competitive market. Antios has raised $96 million in Series B financing to support the ongoing phase 2 development of its hepatitis B drug candidate, ATI-2173.

Gene Silencing 64

Gene Silencing Trials Drugs DNA 64

Delveinsight

AUGUST 6, 2020

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.

Protein 52

Protein Gene Silencing Marketing Gene 52

pharmaphorum

NOVEMBER 24, 2020

The FDA has approved Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder. Patients with PH1 produce far too much oxalate, a substance consumed in food and produced by the body.

FDA Approval 52

FDA Approval Gene Silencing Drugs RNA 52

pharmaphorum

JANUARY 6, 2021

The regulator said it wanted more long-term safety and efficacy data from the REGENERATE study before making a final decision on the drug, which had been expected to be the first entrant into a likely multi-billion dollar market niche. The post High hopes as Inventiva takes NASH contender into phase 3 appeared first on.

Gene Silencing 52

Gene Silencing Regulation Pharma Companies Trials 52

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.

Gene Silencing 52

Gene Silencing RNA Protein Production 52

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

pharmaphorum

JUNE 8, 2022

Zilebesiran (formerly ALN-AGT) is one of a new breed of gene-silencing drugs that are intended to treat common, chronic diseases with infrequent dosing to boost compliance with treatment.

Gene Silencing 52

Gene Silencing Drugs Medicine Regulation 52

pharmaphorum

SEPTEMBER 1, 2021

Novartis’ cholesterol-lowering drug Leqvio will be made available on the NHS in England and Wales, and could help to prevent up to 30,000 deaths, according to NICE.

Drugs 98

Drugs Trials Gene Silencing Genetics 98

Roots Analysis

OCTOBER 22, 2023

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

RNA Protein Genetics Gene Expression 40

The Pharma Data

NOVEMBER 24, 2020

On November 19, the European Commission granted marketing authorization for Oxlumo for the treatment of PH1 in all age groups, following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP). Until today, there were no approved pharmaceutical therapies for PH1.

FDA Approval 52

FDA Approval Production RNA Medicine 52