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

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. . The post Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug appeared first on.

In-Vivo 105

In-Vivo Gene Editing Trials Drugs 105

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 98

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 98

Pharmaceutical Technology

FEBRUARY 20, 2023

In addition to gene therapy, gene silencing, which can be done using ASOs, is also an interesting approach, says Barmada. However, these treatments must target a gene, which is challenging, since many FTD patients lack mutations, says Barmada. Here, ALS and FTD also overlap.

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Development Gene Therapy Protein Gene 246

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 59

Gene Silencing Drugs Medicine RNA 59

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 64

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?

Gene Silencing 97

Gene Silencing Drug Delivery FDA Approval RNA 97

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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Gene Silencing Sales Marketing Drugs 111

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.

Gene Silencing 98

Gene Silencing FDA Approval Trials Marketing 98

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.

FDA Approval 52

FDA Approval Gene Silencing Drugs RNA 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

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.

Gene Silencing 52

Gene Silencing Drugs Medicine Regulation 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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Gene Silencing RNA Protein 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. The post Alnylam finds belly fat gene and drug target in UK Biobank study appeared first on.

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Biobanking Gene Gene Silencing Drugs 52

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

Gene Silencing 52

Gene Silencing Development Drugs Clinical Development 52

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.

Gene Silencing 52

Gene Silencing Regulation Pharma Companies Trials 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.

Gene Silencing 52

Gene Silencing RNA Protein Production 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

XTalks

FEBRUARY 14, 2022

Developed by Orchard Therapeutics, Libmeldy received approval from the European Commission (EC) in December 2020 and about a month later, received a regenerative medicine advanced therapy (RMAT) designation from the US Food and Drug Administration (FDA) to expedite its path to approval in the US.

Gene Therapy 98

Gene Therapy Gene Drugs Gene Silencing 98

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 Gene Silencing Drugs 96

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.

Gene Silencing 98

Gene Silencing Sales Gene Editing Clinical Trials 98

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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Gene Silencing Medicine RNA Licensing 126

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.”

Drugs 98

Drugs Trials Gene Silencing Genetics 98

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.

Trials 59

Trials Gene Silencing RNA Gene Expression 59

Delveinsight

AUGUST 6, 2020

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

Protein 52

Protein Gene Silencing Marketing Gene 52

pharmaphorum

OCTOBER 29, 2020

Earlier this year, the group also signed a licensing deal with Dyno Therapeutics for its AAV capsid-based gene delivery technology, also focused on ocular disease which remains one of Novartis’ core drug development targets despite the spin-off of the Alcon eyecare division in 2019.

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Gene Therapy Gene Gene Silencing Protein 40

Roots Analysis

OCTOBER 22, 2023

In the last few years, researchers have become interested in using in vitro transcribed (IVT) mRNA as a drug delivery agent. Furthermore, these drug candidates can be used to develop precise and individualized therapies that allow patients to produce therapeutic proteins in their own bodies.

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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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FDA Approval Production RNA Medicine 52