NICE recommends gene silencing therapy for porphyria patients on NHS

Pharma Times

Givlaari uses ‘gene silencing’ RNA interference technology, to target the production of pathogenic compounds in people AHP

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

XTalks

Related: Novartis’ $2 Million Gene Therapy Zolgensma Shows ‘Remarkable’ Results and Offers Hope for Children with SMA. A lack of the gene leads to accumulation of porphyrins — essential compounds found in hemoglobin — in the liver.

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New CEO Mortazavi takes UK biotech e-therapeutics into gene silencing

Pharma Phorum

UK biotech e-therapeutics has a new CEO, with executive chairman and former Silence Therapeutics chief Ali Mortazavi chosen to spearhead the next stage in the company’s development into gene silencing and other areas.

Methylation of tRNA-derived fragments regulates gene-silencing activity in bladder cancer

Scienmag

and colleagues have described a novel form of gene regulation that is altered in bladder cancer, leading to the boosting of a gene pathway that helps the cancer cells survive during rapid growth. BIRMINGHAM, Ala. Anindya Dutta, MBBS, Ph.D.,

Alnylam, gene-silencing and biotech in 2020: the pharmaphorum podcast

Pharma Phorum

Alnylam’s Brendan Martin joined the pharmaphorum podcast for episode 23 to talk about his company’s work in gene-silencing and how it could offer a route to target the current coronavirus pandemic.

Novo Nordisk’s gene silencing alliance with Dicerna bears first fruit

Pharma Phorum

Two years after starting to work together, Novo Nordisk and Dicerna have selected the first candidate from a joint project to find new, gene-silencing drugs for liver-related cardiometabolic diseases. News R&D Diabetes Dicerna gene silencing hepatology NASH Novo Nordisk RNAi

FDA Approves First Interchangeable Humira Biosimilar + Gene Silencing Treatment for Porphyria Gets Nod From England’s NICE – Xtalks Life Science Podcast Ep. 34

XTalks

Ayesha also talked about a new gene silencing treatment for porphyria called Givlaari that received recommendation from England’s NICE after having been initially rejected by the health watchdog last year.

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

Scienmag

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. Their findings, reported in Nature Structural and Molecular Biology, also explain certain mysteries about the […]. Medicine & Health Biology cancer Genetics Molecular Biology

Amgen eyes swift move to ph3 for cardiovascular drug olpasiran

Pharma Phorum

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.

NICE backs NHS use of Novartis’ cholesterol drug Leqvio

Pharma Phorum

Market Access News cholesterol gene silencing HEOR inclisiran Leqvio market access NHS NICE Novartis RNAiNovartis’ 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.

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

Pharma Phorum

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 News R&D Dicerna gene silencing M&A Novo Nordisk RNAi

RNA 52

Cash round sets up trial of InteRNA’s microRNA for cancer

Pharma Phorum

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. Dutch biotech InteRNA Technologies has raised €18.5

RNA 56

Three-in-one approach boosts the silencing power of CRISPR

Scienmag

A newly developed CRISPR-Cas9-based tool carries out efficient and long-term gene silencing by epigenetic editing Credit: BioDesign Research Originally discovered as a bacterial mode of defense against invading viruses, the remarkable ability of CRISPR-Cas9 to modify specific locations of DNA has made it a researcher favorite among gene editing tools. Technology and Engineering Biology Biotechnology Cell Biology Gene Therapy Genes Genetics Molecular Biology

Mathur studying cytosolic access and instability of DNA nanoparticles

Scienmag

A number of candidate therapies such as CRISPR-Cas9 and gene silencing require the efficient delivery of functional nucleic acids to the cell cytosol and nucleus. Divita Mathur, Research Assistant Professor, is studying cytosolic access and instability of DNA nanoparticles. Unfortunately, such therapies currently lack proper delivery mechanisms, precluding their widespread applicability. Self-assembled deoxyribonucleic acid (DNA) […]. Biology Genetics

A new strategy for siRNA stabilization by an artificial cationic oligosaccharide

Scienmag

Credit: TMIMS RNA interference is a gene regulatory mechanism in which the expression of specific genes is downregulated by endogenous microRNAs or by small interfering RNAs (siRNAs).

Novartis keeps a close eye on gene therapy with Vedere Bio buy

Pharma Phorum

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. Incubated by Atlas Ventures, Cambridge, Massachusetts Vedere has been flying under the radar since it was founded in 2019 to develop adeno-associated virus (AAV) based gene therapies for ocular diseases, but didn’t escape the beady eye of Novartis.

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

XTalks

Related: Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data. Libmeldy is a lentiviral vector-based gene therapy that delivers working copies of the ARSA gene that is mutated in MLD.

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

Pharma Phorum

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.

Alnylam gets NICE backing for porphyria therapy Givlaari

Pharma Phorum

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.

Innovate UK funding for new precision cancer treatment technology

Pharma Phorum

Programmable Oligonucleotide Delivery System (PODS), developed by Sixfold Bioscience, is a versatile system which delivers short interfering RNA (siRNA) gene silencing cargo to specific cancer cells.

Novo Nordisk forges close ties with biotech VC Flagship

Pharma Phorum

billion takeover of gene-silencing specialist Dicerna Pharma last year, another deal aimed at extending its R&D capabilities by bolting on a new platform technology – in this case RNA interference (RNAi) drugs.

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

XTalks

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.

DNA 79

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

XTalks

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. It’s a great tool for controlling gene expression.”.

DNA 52

The 3rd Annual RNAi – Based Therapeutics Summit Returns!

Pharma Phorum

Explore latest progress in siRNA chemistry to increase activity, stability, specificity & potential off target effects to improve cellular targeting with insights from Silence Therapeutics & Aro Biotherapeutics.

MHRA gives Alnylam ‘innovation passport’ for hypertension drug zilebesiran

Pharma Phorum

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.

Novo Nordisk bulks up in rare diseases with $1.2bn Prothena deal

Pharma Phorum

In 2019, for example it agreed to work with Dicerna on gene-silencing for liver-related cardio-metabolic disease in a deal that involved a $225 million upfront payment.

Race for hepatitis B drug intensifies as Antios raises $96m for phase 2 trial

Pharma Phorum

It’s the chronic form of the disease that is the main target, with Ionis is working with GSK on an antisense drug that can suppress the hepatitis B virus (HBV), and Roche is working with Dicerna on a rival gene silencing drug.

Two biotechs target dry eye disease after Novartis’ Xiidra problems

Pharma Phorum

Tivanisiran is based on gene silencing technology using RNA interference (RNAi) and selectively inhibits production of the transient receptor potential cation channel (TRPV1).

Novartis’ big bet on inclisiran nears fruition, as CHMP backs drug

Pharma Phorum

If approved, inclisiran would become the first and only gene-silencing drug to reduce low-density lipoprotein cholesterol (LDL-C) in these patients.

High hopes as Inventiva takes NASH contender into phase 3

Pharma Phorum

Only this morning, Novo Nordisk selected the first candidate from a project with Dicerna to find new gene-silencing drugs to treat liver-related diseases including NASH.

FDA approves Alnylam’s ultra-rare disease drug Oxlumo

Pharma Phorum

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.

Unfolding The Folds Of Transthyretin

Delveinsight

Hereditary ATTR amyloidosis , also known as Familial ATTR, is an autosomal dominant disease and a result of mutations in the TTR gene, thus it is passed down from one generation to the next one. Gene-silencing Therapies.

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

Pharma Marketing Network

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.

Scopus BioPharma Announces Pricing of $9 Million Follow-On Public Offering

The Pharma Data

The company’s lead drug candidate is a novel, targeted immuno-oncology gene therapy for the treatment of multiple cancers. The company’s lead drug candidate is a novel, targeted immuno-oncology gene therapy for the treatment of multiple cancers. NEW YORK , Jan.

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

Pharma Phorum

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.

Novartis’ big bet on inclisiran nears fruition, as CHMP backs drug

Pharma Phorum

If approved, inclisiran would become the first and only gene-silencing drug to reduce low-density lipoprotein cholesterol (LDL-C) in these patients.

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

Pharma Phorum

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.”.

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

The Pharma Data

About RNAi RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1.