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

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.

Gene Silencing

Gene Silencing Gene RNA 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

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. — Eric Topol (@EricTopol) June 26, 2021.

In-Vivo

In-Vivo Gene Editing Trials Drugs

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. Epigenetic Editing with CRISPR.

DNA

DNA Gene Gene Silencing Genomics

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

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 billion in cash.

Gene Silencing

Gene Silencing RNA Protein Gene

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.

FDA Approval

FDA Approval Gene Silencing Drugs RNA

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.

Biobanking

Biobanking Gene Gene Silencing Drugs

Using CRISPR to Edit the Epigenome Might Be Easier Than We Thought

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. These modifications regulate gene expression without altering the sequence or structure of DNA.

DNA

DNA Gene Gene Silencing Genomics

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

pharmaphorum

DECEMBER 23, 2020

In summer, Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder Oxlumo was approved in the European Union and the US. It was the third approval from Alnylam’s pipeline of RNA interference therapeutics to make it to market.

Marketing

Marketing Gene Gene Silencing Drugs

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.

Protein

Protein Gene Silencing Marketing Gene

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 works by degrading HAO1 messenger RNA and reducing the synthesis of GO, which inhibits hepatic production of oxalate – the toxic metabolite responsible for the clinical manifestations of PH1. Oxlumo should be administered by a healthcare professional.

FDA Approval

FDA Approval Production RNA Medicine

Clinical Research Informer

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

Alnylam gets NICE backing for porphyria therapy Givlaari

Trending Sources

RNA Therapeutics: A Novel Approach to Treating Diseases

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

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

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

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

Amgen eyes swift move to ph3 for cardiovascular drug olpasiran

FDA approves Alnylam’s ultra-rare disease drug Oxlumo

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

Using CRISPR to Edit the Epigenome Might Be Easier Than We Thought

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

Unfolding The Folds Of Transthyretin

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

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