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

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., 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.

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

Drug Discovery World

SEPTEMBER 5, 2022

Knockout’ mice are bred in a laboratory with specific genes silenced or ‘knocked out’. The goal of this final phase is to produce and phenotype hundreds of knockout mouse models for genes with little to no known function, to better understand the genetic bases for diseases in humans and animals. . Institutions in 15 countries.

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Gene Silencing Gene Genetics Genome 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.

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Gene Silencing RNA Production Protein 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. Alnylam licensed inclisiran to The Medicines Company, which was bought last year by Novartis for $9.7

FDA Approval 52

FDA Approval Gene Silencing Drugs RNA 52

pharmaphorum

OCTOBER 19, 2020

Novartis’ near-$10 billion takeover of The Medicines Company last year was focused mainly on one asset – cholesterol-lowering drug inclisiran – and the Swiss pharma is now a step closer to getting a return on its investment.

Drugs 98

Drugs Gene Silencing Sales Antibody 98

pharmaphorum

DECEMBER 13, 2020

Alexion has been working hard to flesh out is pipeline as well, snapping up Achillion Pharma, Syntimmune, Wilson Therapeutics and Portola and forging an alliance with gene-silencing specialist Dicerna focusing on complement diseases.

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Gene Silencing Sales Marketing Drugs 111

pharmaphorum

OCTOBER 19, 2020

Novartis’ near-$10 billion takeover of The Medicines Company last year was focused mainly on one asset – cholesterol-lowering drug inclisiran – and the Swiss pharma is now a step closer to getting a return on its investment.

Drugs 52

Drugs Gene Silencing Sales Antibody 52

pharmaphorum

DECEMBER 23, 2020

But there was some considerable progress in other fields of medicine even though research efforts were diverted away, reports Richard Staines. It has been a year dominated by the pandemic and many life sciences research projects were put on hold as big pharma turned its attention to vaccines and therapies. Rare disease progress.

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Marketing Gene Silencing Gene Drugs 97

Drug Discovery World

OCTOBER 4, 2022

Cell and gene therapies. There’s no question that cell and gene therapies have emerged as some of the most promising and innovative medicines on the market. . With 3,633 advanced therapeutical medicinal products (ATMPs) currently in some form of clinical or pre- clinical development, the sector shows no signs of slowing down.

Gene Therapy 52

Gene Therapy Drugs Gene Manufacturing 52

Drug Discovery World

JULY 25, 2023

The results are published in the New England Journal of Medicine (NEJM). In this context, the data we have published in NEJM are exciting, suggesting the potential role for zilebesiran to treat hypertension in a novel way via a novel, subcutaneously administered gene silencing approach to hypertension.”

Gene Silencing 97

Gene Silencing Clinical Development Gene Medicine 97

pharmaphorum

JUNE 8, 2022

The UK medicines regulator has awarded Alnylam’s RNAi-based therapy zilebesiran for hypertension an ‘innovation passport ‘, a designation designed to speed up NHS access to promising new medicines. The study is enrolling adults whose blood pressure is not adequately controlled by standard medicines.

Gene Silencing 52

Gene Silencing Drugs Medicine Regulation 52

XTalks

FEBRUARY 14, 2022

Developed by Orchard Therapeutics, Libmeldy received approval from the European Commission (EC) in December 2020 and about a month later, received a regenerative medicine advanced therapy (RMAT) designation from the US Food and Drug Administration (FDA) to expedite its path to approval in the US. million (approximately $3.8 million USD).

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Gene Therapy Drugs Gene Gene Silencing 98

pharmaphorum

JUNE 28, 2021

The reductions matched the efficacy of current therapies for ATTR amyloidosis that require chronic dosing such as Alnylam’s Onpattro (patisiran) and Ionis/Akcea’s Tegsedi (inotersen) – both gene-silencing agents which can cost around $450,000 a year. Regeneron has partnered the company since 2016.

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In-Vivo Gene Editing Trials Drugs 105

Drug Discovery World

AUGUST 1, 2023

The NIH states: “This natural mechanism for sequence-specific gene silencing promises to revolutionise experimental biology and may have important practical applications in functional genomics, therapeutic intervention, agriculture and other areas.” There are many examples of ways in which RNAi is impacting drug discovery.

RNA 52

RNA Drugs Gene Silencing Vaccination 52

pharmaphorum

MARCH 4, 2021

Tivanisiran is based on gene silencing technology using RNA interference (RNAi) and selectively inhibits production of the transient receptor potential cation channel (TRPV1). The design of the protocol for the trial and its authorization are based on the scientific evidence of safety and efficacy obtained in the phase 3 HELIX trial.

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Gene Silencing FDA Approval Trials Marketing 98

The Pharma Data

APRIL 27, 2023

These early results establish the first human translation of Alnylam’s proprietary C16-siRNA conjugate platform for central nervous system (CNS) delivery and are the first clinical demonstration of gene silencing in the human brain using an RNAi therapeutic.

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Gene Silencing Protein Gene Engineer 40

The Pharma Data

NOVEMBER 24, 2020

Results from the ILLUMINATE-A and ILLUMINATE-B studies demonstrate that Oxlumo addresses the underlying pathophysiology of PH1 in adults, children and infants, and we believe this newly approved medicine has the potential to change the course of this progressive disease,” said Akshay Vaishnaw, M.D., President of R&D at Alnylam.

FDA Approval 52

FDA Approval Production RNA Medicine 52

Roots Analysis

OCTOBER 22, 2023

RNA interference (RNAi) Therapeutics : Fundamentally, RNAi is a natural process of post-transcriptional gene silencing, involving short strands of nucleic acids. Cells use this process to silence and / or inhibit gene expression, via targeted degradation of specific (unwanted) mRNA molecules.

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RNA Protein Genetics Gene Expression 40

Drug Discovery World

JANUARY 23, 2023

A second generation CRISPR-Cas gene editing system, base editing, reduces the risk of potentially deleterious effects associated with other gene editing technologies, presenting a safe and effective means of editing cells for therapeutic applications.

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Gene Editing Gene Engineer DNA 52