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.

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Gene Silencing Drugs Medicine RNA 57

pharmaphorum

SEPTEMBER 23, 2020

Sixfold Bioscience in partnership with Medicines Discovery Catapult, has been given the green light, through the award of an Innovate UK Smart Grant to test a pioneering system designed to deliver drugs directly to cancerous cells, without impacting the healthy cells around it.

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Drug Delivery Systems Gene Silencing Drug Delivery Medicine 141

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.

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Gene Silencing Gene RNA Drugs 96

pharmaphorum

OCTOBER 29, 2020

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. Novartis’ big move in the gene therapy market came when it bought AveXis for $8.7 It also gets two preclinical-stage development projects.

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Gene Therapy Gene Gene Silencing Protein 40

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. That said, Novartis is no stranger to building a slow-starting cardiovascular drug into a blockbuster franchise.

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Drugs Gene Silencing Sales Antibody 96

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. 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. .

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Gene Therapy Drugs Gene Manufacturing 52

pharmaphorum

APRIL 13, 2021

Antios has raised $96 million in Series B financing to support the ongoing phase 2 development of its hepatitis B drug candidate, ATI-2173. The post Race for hepatitis B drug intensifies as Antios raises $96m for phase 2 trial appeared first on. Feature image courtesy of the US Centers for Disease Control.

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Gene Silencing Trials Drugs DNA 62

XTalks

JUNE 14, 2023

percent received approval from the US Food and Drug Administration (FDA) to treat dry eye disease. Attendees will understand best practices for novel drug delivery design and development. Attendees will understand best practices for novel drug delivery design and development. What Is Dry Eye Disease?

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Gene Silencing Drug Delivery FDA Approval RNA 97

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

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 is hoping that revenue will stream from its new products and payments from Novartis, which owns the rights to cholesterol lowering drug inclisiran.

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FDA Approval Gene Silencing Drugs RNA 52

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.

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pharmaphorum

MAY 11, 2022

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. The move follows Novo Nordisk’s $3.3

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pharmaphorum

FEBRUARY 9, 2021

million ($22 million) in second-round financing as it prepares to start clinical trials of its lead drug candidate INT-1B3 for solid tumours. The start of dosing in the phase 1/1b trial of the drug has been delayed by the COVID-19 pandemic, but recruitment of the target 80 participants with advanced solid tumours is reported to be ongoing.

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Trials Gene Silencing RNA Gene Expression 57

pharmaphorum

JULY 13, 2021

Diabetes giant Novo Nordisk has opened a new front in its rare disease R&D, thanks to an acquisition agreement with Prothena focusing on drugs for ATTR amyloidosis. 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.

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pharmaphorum

FEBRUARY 18, 2022

With 28 pioneering speakers from large pharma, innovative biotech and KOLs of academia who are ready to discuss the full and comprehensive range of RNAi drugs from discovery to development and beyond, join us in Boston to hear how they address the major challenges facing the industry. Realize the full potential of RNAi carriers 2.0

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Gene Silencing Development Drugs Clinical Development 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. That said, Novartis is no stranger to building a slow-starting cardiovascular drug into a blockbuster franchise.

Drugs 52

Drugs Gene Silencing Sales Antibody 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

SEPTEMBER 1, 2021

Novartis’ 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. Professor Kausik Ray of Imperial College London said that the drug is a game-changer because of the drug’s “convenience, safety and duration of effect.”

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Drugs Trials Gene Silencing Genetics 94

Pharmaceutical Technology

JANUARY 31, 2023

Gene therapy company uniQure has entered into a global licensing agreement with Apic Bio for APB-102 to treat patients with amyotrophic lateral sclerosis (ALS) caused by mutations in superoxide dismutase 1 (SOD1). APB-102 is designed to be a one-time, intrathecally administered gene therapy for ALS patients.

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pharmaphorum

JANUARY 6, 2021

Analysts are continuing to back Inventiva’s lanifibranor as a potential “best-in-class” drug for the fatty liver disease NASH, as the company prepares for the launch of a phase 3 trial in spring. There are no approved drugs for NASH – full name non-alcoholic steatohepatitis – after the FDA rejected Intercept’s obeticholic acid last year.

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Gene Silencing Regulation Pharma Companies Doctors 52

The Pharma Data

JANUARY 26, 2021

The company’s lead drug candidate is a novel, targeted immuno-oncology gene therapy for the treatment of multiple cancers. Scopus intends to use the proceeds of the offering principally for further development of the company’s lead drug candidate, including in combination with checkpoint inhibitors.

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Gene Therapy Gene Silencing Gene Containment 52

pharmaphorum

DECEMBER 13, 2020

The deal – the largest in the pharma sector since the start of the pandemic – bolsters AZ’s immunology franchise with $4 billion blockbuster Soliris (eculizumab) and longer acting follow-up Ultomiris (ravulizumab), plus a pipeline of 11 drugs for rare and autoimmune diseases.

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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.

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Marketing Gene Silencing Gene Drugs 74

Delveinsight

AUGUST 6, 2020

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. The present hATTR market comprises only a few approved drugs , which maintain the monopoly in the market.

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Gene Silencing Protein Marketing Gene 52

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. Transgenic mice in which the gene has been deleted have improved control of blood glucose and insulin sensitivity.

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Biobanking Gene Gene Silencing Drugs 52

Pharmaceutical Technology

FEBRUARY 20, 2023

Different approaches that are studied include antisense oligonucleotides (ASOs), and gene therapies, which are in early clinical trials. The therapy is specifically aimed at treating FTD patients who have mutations in their granulin gene, which encodes for the protein progranulin (PGRN). This protein promotes lysosomal function.

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Development Gene Therapy Protein Gene 246

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%.

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Drug Discovery World

JULY 25, 2023

“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.”

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pharmaphorum

JUNE 8, 2022

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. The post MHRA gives Alnylam ‘innovation passport’ for hypertension drug zilebesiran appeared first on.

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Gene Silencing Drugs Medicine Regulation 52

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.

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RNA Drugs Gene Silencing Vaccination 52

pharmaphorum

MARCH 4, 2021

The pipeline for dry eye disease drugs is beginning to heat up, with two biotechs announcing key developments, aiming to disrupt a market led by Allergan and Novartis. billion for its FDA-approved dry eye drug Xiidra during the Japanese pharma’s merger with Shire in 2019, outlining the sales potential in this market niche.

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Gene Silencing FDA Approval Trials Marketing 98

Drug Discovery World

JANUARY 23, 2023

Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies. Adult fibroblasts were cultured in a cocktail of transcription factors to reactivate embryonic gene expression patterns 2.

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Gene Editing Gene Engineer DNA 52

Roots Analysis

OCTOBER 22, 2023

Coding RNAs include messenger RNA (mRNA) and short interfering RNA (siRNA), which encode proteins and silence gene expression, respectively. In the last few years, researchers have become interested in using in vitro transcribed (IVT) mRNA as a drug delivery agent. RNA therapeutics offer several advantages over small molecules.

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RNA Protein Genetics Gene Expression 40

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. Injection site reactions (ISRs) were the most common drug-related adverse reaction.

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