Gene Silencing, Genetics and Medicine - Clinical Research Informer

Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data

XTalks

OCTOBER 26, 2021

After initial rejection from the National Institute for Health and Care Excellence (NICE) last year, the non-departmental public body of the Department of Health in England has now given the green light to the gene silencing treatment Givlaari (givosiran) for the treatment of the rare metabolic disorder, acute intermittent porphyria (AIP).

Gene Silencing

Gene Silencing Gene Clinical Trials Clinical Trials

Study resolves long-running controversy over critical step in gene silencing

Scienmag

JANUARY 4, 2021

BOSTON – A long-running debate over how an important gene-silencing protein identifies its targets has been resolved by researchers at Massachusetts General Hospital (MGH). The findings could yield important implications for development of drugs to treat cancer and other diseases.

Gene Silencing

Gene Silencing Gene Protein Development

Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem

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

Genetics Medicine Gene Therapy Marketing

Alnylam gets NICE backing for porphyria therapy Givlaari

pharmaphorum

OCTOBER 21, 2021

UK cost-effectiveness watchdog NICE is set to recommend NHS use of Alnylam’s gene-silencing therapy Givlaari in England and Wales for the rare disease acute hepatic porphyria (AHP), after its advisors issued a positive verdict on the drug. It was approved by the European Medicines Agency (EMA) last year.

Gene Silencing

Gene Silencing Gene RNA Drugs

NIH renews knockout mouse project for 3rd time

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. .

Gene Silencing

Gene Silencing Gene Genetics Genome

Amgen eyes swift move to ph3 for cardiovascular drug olpasiran

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

Gene Silencing Drugs Medicine RNA

FDA approves Alnylam’s ultra-rare disease drug Oxlumo

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

FDA Approval Gene Silencing Drugs RNA

Alexion finally has a buyer – and it’s AstraZeneca with $39bn on the table

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.

Gene Silencing

Gene Silencing Sales Marketing Drugs

The trends driving ELRIG Drug Discovery 2022

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

Gene Therapy Drugs Gene Manufacturing

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

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.

Marketing

Marketing Gene Silencing Gene Drugs

UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD

XTalks

FEBRUARY 14, 2022

The UK’s National Health Service (NHS) has recommended the use of Libmeldy for the treatment of the rare genetic disease metachromatic leukodystrophy (MLD). Related: Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data. million (approximately $3.8 million USD).

Gene Therapy

Gene Therapy Drugs Gene Gene Silencing

Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug

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.

In-Vivo

In-Vivo Gene Editing Trials Drugs

World RNA Day: What impact RNA it had on drug discovery?

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

RNA Drugs Gene Silencing Vaccination

RNA Therapeutics: A Novel Approach to Treating Diseases

Roots Analysis

OCTOBER 22, 2023

RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. These therapeutics are broadly classified into two categories, namely coding RNAs and non-coding RNAs.

RNA

RNA Protein Genetics Gene Expression

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1

The Pharma Data

NOVEMBER 24, 2020

PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. Oxlumo marks our third FDA approval in less than three years, positioning us to meet or exceed our Alnylam 2020 strategy and goals, and further highlighting the productivity of our RNAi platform and the speed at which we can bring innovative medicines to patients.

FDA Approval

FDA Approval Production RNA Medicine

REGENERON AND ALNYLAM REPORT POSITIVE INTERIM PHASE 1

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.

Gene Silencing

Gene Silencing Protein Gene Engineer

The use of base editing in stem-cell based therapies

Drug Discovery World

JANUARY 23, 2023

Increasing interest in CRISPR-Cas gene editing systems over recent years has delivered innovative developments in precision genome engineering technologies, holding enormous potential for applications across the healthcare sector. Single-cell RNA-sequencing of differentiating iPS cells reveals dynamic genetic effects on gene expression.

Gene Editing

Gene Editing Gene Engineer DNA

Clinical Research Informer

Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data

Study resolves long-running controversy over critical step in gene silencing

Trending Sources

Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem

Alnylam gets NICE backing for porphyria therapy Givlaari

NIH renews knockout mouse project for 3rd time

Amgen eyes swift move to ph3 for cardiovascular drug olpasiran

FDA approves Alnylam’s ultra-rare disease drug Oxlumo

Alexion finally has a buyer – and it’s AstraZeneca with $39bn on the table

The trends driving ELRIG Drug Discovery 2022

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

UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD

Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug

World RNA Day: What impact RNA it had on drug discovery?

RNA Therapeutics: A Novel Approach to Treating Diseases

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1

REGENERON AND ALNYLAM REPORT POSITIVE INTERIM PHASE 1

The use of base editing in stem-cell based therapies

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