BioTech 365

MARCH 10, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Northwestern U’s novel gene-silencing RNA drug shows promise in glioblastoma.Northwestern U’s novel gene-silencing RNA drug shows promise in glioblastoma aliu Wed, 03/10/2021 – 06:56 from FierceBiotech: Biotech (..)

Gene Silencing 40

Gene Silencing RNA Gene Drugs 40

Scienmag

JANUARY 4, 2021

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.

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Gene Silencing Gene Protein Development 41

Pharmaceutical Technology

JANUARY 31, 2023

Under the terms of the deal, uniQure will obtain the worldwide rights to develop and market the clinical stage gene therapy, APB-102. uniQure stated that the license of APB-102 further strengthens its gene therapies pipeline developed for the treatment of neurological disorders as well as miRNA-based gene silencing programmes.

Gene Therapy 277

Gene Therapy Licensing Licensing Gene 277

Scienmag

MAY 9, 2022

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., Credit: UAB BIRMINGHAM, Ala. Anindya Dutta, MBBS, Ph.D.,

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Gene Silencing Regulation Gene 61

XTalks

OCTOBER 27, 2021

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

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Gene Silencing Life Science FDA Approval Gene 40

pharmaphorum

SEPTEMBER 23, 2020

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.

Drug Delivery Systems 141

Drug Delivery Systems Gene Silencing Drug Delivery Medicine 141

Scienmag

JULY 2, 2021

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. (..)

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Gene Silencing Gene Editing Gene DNA 75

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 98

Gene Silencing Gene RNA Drugs 98

Scienmag

OCTOBER 16, 2020

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). Although siRNAs have broad potential for gene-silencing therapy, their instability is one of the difficulties to develop siRNA-based agents.

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Gene Silencing RNA Gene Development 57

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.

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Development Gene Therapy Protein Gene 246

Scienmag

JUNE 7, 2021

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.

DNA 51

DNA Gene Silencing Gene Research 51

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

XTalks

MAY 4, 2021

The tool could also prove to be safer than conventional CRISPR-based gene therapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. Genes can be switched on with a complementary tool called CRISPRon that has also been described in the paper.

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DNA Gene Gene Silencing Genome 98

XTalks

JUNE 14, 2023

Tivanisiran is a preservative-free eye drop that uses RNAi technology, specifically gene silencing, to target and control the signs and symptoms of dry eye disease. It is a small synthetic double-stranded RNA oligonucleotide (siRNA) designed for this purpose, representing the state-of-the-art in gene silencing technology.

Gene Silencing 97

Gene Silencing Drug Delivery FDA Approval RNA 97

pharmaphorum

JULY 13, 2021

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. There has been an increase in partnering activity at Novo Nordisk as it follows a diversification strategy.

Gene Silencing 98

Gene Silencing Sales Gene Editing Clinical Trials 98

pharmaphorum

OCTOBER 19, 2020

If approved, inclisiran would become the first and only gene-silencing drug to reduce low-density lipoprotein cholesterol (LDL-C) in these patients. The drug inhibits PCSK9 – the same target as Amgen’s Repatha (evolocumab) and Sanofi/Regeneron’s Repatha (alirocumab) – but is dosed only twice a year rather than every month.

Drugs 98

Drugs Gene Silencing Sales Antibody 98

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.

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In-Vivo Gene Editing Trials Drugs 105

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 59

pharmaphorum

MARCH 4, 2021

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

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Gene Silencing FDA Approval Trials Marketing 98

pharmaphorum

DECEMBER 13, 2020

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.

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Gene Silencing Sales Marketing Drugs 111

pharmaphorum

APRIL 13, 2021

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.

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Gene Silencing Trials Drugs DNA 64

pharmaphorum

FEBRUARY 9, 2021

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.

Trials 59

Trials Gene Silencing RNA Gene Expression 59

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.

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Gene Silencing RNA Protein Gene 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.

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Gene Silencing Development Drugs Clinical Development 52

XTalks

FEBRUARY 14, 2022

Related: Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data. It is an autosomal recessive disorder, which means that if both parents are carriers of the faulty gene that causes the disease, there is a one in four chance that their child will develop the disease.

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Gene Therapy Gene Drugs Gene Silencing 98

pharmaphorum

JANUARY 6, 2021

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. A host of other pharma companies including Gilead, Novo Nordisk, Merck & Co are also developing potential NASH drugs.

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Gene Silencing Regulation Pharma Companies Trials 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.

FDA Approval 52

FDA Approval Gene Silencing Drugs RNA 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

XTalks

MAY 4, 2021

The tool could also prove to be safer than conventional CRISPR-based gene therapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. Genes can be switched on with a complementary tool called CRISPRon that has also been described in the paper.

DNA 52

DNA Gene Gene Silencing Genome 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.

Gene Silencing 52

Gene Silencing Drugs Medicine Regulation 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.

Biobanking 52

Biobanking Gene Gene Silencing Drugs 52

pharmaphorum

OCTOBER 19, 2020

If approved, inclisiran would become the first and only gene-silencing drug to reduce low-density lipoprotein cholesterol (LDL-C) in these patients. The drug inhibits PCSK9 – the same target as Amgen’s Repatha (evolocumab) and Sanofi/Regeneron’s Repatha (alirocumab) – but is dosed only twice a year rather than every month.

Drugs 52

Drugs Gene Silencing Sales Antibody 52

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 past years have witnessed a shift from off-label and symptomatic therapies to approved TTR stabilizers and gene-silencing therapies.

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.

Gene Therapy 40

Gene Therapy Gene Gene Silencing Protein 40

The Pharma Data

JANUARY 26, 2021

This drug candidate is highly distinctive, encompassing both gene therapy and immunotherapy by synthetically linking siRNA to an oligonucleotide TLR9 agonist, creating the potential for targeted gene silencing with simultaneous TLR stimulation and immune activation in the tumor microenvironment.

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Gene Therapy Gene Silencing Gene Containment 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.

Drugs 98

Drugs Trials Gene Silencing Genetics 98

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. Rare disease progress.

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Marketing Gene Gene Silencing Drugs 85