Drugs, Gene Silencing, Genetics and RNA - 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

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

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

Novo Nordisk snaps up RNAi partner Dicerna in $3.3bn takeover deal

pharmaphorum

NOVEMBER 18, 2021

Danish drugmaker Novo Nordisk must like what it has seen in its two-year-old alliance with gene-silencing specialist Dicerna Pharma – it has just agreed to acquire the biotech for $3.3 Dicerna has been a hot tip for takeover for a couple of years, as the list of approved drugs that work via RNAi has lengthened. billion in cash.

Gene Silencing

Gene Silencing RNA Protein Gene

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. The post FDA approves Alnylam’s ultra-rare disease drug Oxlumo appeared first on.

FDA Approval

FDA Approval Gene Silencing Drugs RNA

The trends driving ELRIG Drug Discovery 2022

Drug Discovery World

OCTOBER 4, 2022

DDW Editor Reece Armstrong explores some of the trends that will be featured in sessions throughout ELRIG Drug Discovery 2022 and the news behind them. Automated systems have become a prominent feature throughout drug discovery, helping scientists save time and assisting in reproducible experiments for more verifiable results.

Gene Therapy

Gene Therapy Drugs Gene Manufacturing

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

Drug Discovery World

AUGUST 1, 2023

1 August was first chosen as World RNA Day in 2018 as a play on AUG (adenine, uracil and guanine), a triple sequence of RNA (called a codon) that initiates protein synthesis by the cell. Though some genetic mutations are harmful, others can be protective.

RNA

RNA Drugs Gene Silencing Vaccination

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

pharmaphorum

DECEMBER 23, 2020

The results also suggest further uses for the drug in early disease and more revenues to come from one of the company’s biggest success stories from the last decade. Unlike conventional cancer drugs Vitravki can be used in tumours with this mutation regardless of whether they are in the body.

Marketing

Marketing Gene Silencing Gene Drugs

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. Non-coding RNAs include antisense oligonucleotides (ASOs) and RNA aptamers.

RNA

RNA Protein Genetics Gene Expression

Unfolding The Folds Of Transthyretin

Delveinsight

AUGUST 6, 2020

The hATTR diagnosis is mainly dependent on symptoms and can be confirmed via performing tissue biopsies, genetic testing, and imaging studies. Gene-silencing Therapies. Silencing or modifying gene TTR aims to reduce the effect of both variant and wild-type TTR gene, thus reducing hepatic production by targeting its mRNA.

Gene Silencing

Gene Silencing Protein Marketing Gene

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

pharmaphorum

JUNE 28, 2021

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . — Eric Topol (@EricTopol) June 26, 2021. In three more patients treated at 0.1mg/kg levels fell by 52%.

In-Vivo

In-Vivo Gene Editing Trials Drugs

Alnylam finds belly fat gene and drug target in UK Biobank study

pharmaphorum

JULY 28, 2022

Alnylam Pharma has made a name for itself, developing gene-silencing therapies for rare disorders, but its latest discovery could take it into a much larger category – metabolic and cardiovascular disease. The post Alnylam finds belly fat gene and drug target in UK Biobank study appeared first on.

Biobanking

Biobanking Gene Gene Silencing Drugs

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

The Pharma Data

NOVEMBER 24, 2020

Food and Drug Administration (FDA) approved Oxlumo (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients. PH1 is an ultra-rare genetic disease characterized by oxalate overproduction.

FDA Approval

FDA Approval Production RNA Medicine

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

Amgen eyes swift move to ph3 for cardiovascular drug olpasiran

Trending Sources

Alnylam gets NICE backing for porphyria therapy Givlaari

Novo Nordisk snaps up RNAi partner Dicerna in $3.3bn takeover deal

FDA approves Alnylam’s ultra-rare disease drug Oxlumo

The trends driving ELRIG Drug Discovery 2022

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

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

RNA Therapeutics: A Novel Approach to Treating Diseases

Unfolding The Folds Of Transthyretin

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

Alnylam finds belly fat gene and drug target in UK Biobank study

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

The use of base editing in stem-cell based therapies

Stay Connected