NICE recommends gene silencing therapy for porphyria patients on NHS
Pharma Times
OCTOBER 21, 2021
Givlaari uses ‘gene silencing’ RNA interference technology, to target the production of pathogenic compounds in people AHP
This site uses cookies to improve your experience. By viewing our content, you are accepting the use of cookies. To help us insure we adhere to various privacy regulations, please select your country/region of residence. If you do not select a country we will assume you are from the United States. View our privacy policy and terms of use.
Pharma Times
OCTOBER 21, 2021
Givlaari uses ‘gene silencing’ RNA interference technology, to target the production of pathogenic compounds in people AHP
BioPharma Reporter
OCTOBER 2, 2023
Laverock Therapeutics, the gene editing-induced gene silencing platform for human therapeutic applications, has expanded its seed funding round to Â13.5 million.
This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.
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).
Bio Pharma Dive
JULY 17, 2023
For $500 million, Novartis will acquire DTx Pharma and its preclinical neurological disease drugs, marking the Swiss company’s latest investment in gene-silencing medicines.
pharmaphorum
OCTOBER 13, 2020
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. The post New CEO Mortazavi takes UK biotech e-therapeutics into gene silencing appeared first on.
pharmaphorum
AUGUST 27, 2020
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. The post Alnylam, gene-silencing and biotech in 2020: the pharmaphorum podcast appeared first on.
pharmaphorum
JANUARY 6, 2021
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. The post Novo Nordisk’s gene silencing alliance with Dicerna bears first fruit appeared first on.
BioSpace
JUNE 16, 2024
The plethora of genes involved in obesity presents an intriguing opportunity for both gene silencing and ex vivo gene therapy approaches.
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 (..)
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.
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.
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.,
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. (..)
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.
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.
Drug Discovery World
JULY 24, 2024
ASOs work by stopping a gene, in this case the mutated TUBB4A gene, from forming toxic proteins, so it is a type of gene-silencing. A mutated TUBB4A gene results ultimately in less myelin for insulating nerve fibres in the brain, leading to disruption of the signals between neurons.
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.
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.
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.
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.
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
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.
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.
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.
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.
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. .
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.”
Pharma Marketing Network
DECEMBER 21, 2020
For example, “gene silencing” is a less-than-ideal description for the mechanism of RNA interference (RNAi) therapeutics. Although this approach “silences” the output of genes (by disrupting messenger RNA [mRNA]), the lack of specificity suggests the possibility of direct action on genes in the nucleus.
Expert insights. Personalized for you.
We have resent the email to
Are you sure you want to cancel your subscriptions?
Let's personalize your content