Scienmag

MARCH 17, 2022

Cells use RNA as a versatile tool to regulate the activity of their genes. Small snippets of RNA can fine-tune how much protein is produced from various genes; some small RNAs can shut genes off altogether.

RNA 76

RNA Gene Protein Regulation 76

XTalks

OCTOBER 8, 2024

The 2024 Nobel Prize in Physiology or Medicine has been awarded to American scientists Victor Ambros and Gary Ruvkun for their groundbreaking discovery of microRNA (or miRNA) and its role in post-transcriptional gene regulation. A gene contains instructions within our DNA.

Regulation 52

Regulation Genetics Scientist RNA 52

Scienmag

MARCH 10, 2021

In this article the authors Jingdun Xie, Zhenhua Qi, Xiaolin Luo, Fang Yan, Wei Xing, Weian Zeng, Dongtai Chen and Qiang Li; from Sun Yat-sen University, Guangzhou, Guangdong, China discuss integration analysis of m6A regulators and m6A-related genes in hepatocellular carcinoma (HCC). N6-Methyladenosine (m6A) RNA […].

Regulation 44

Regulation Gene RNA Medicine 44

The Pharma Data

JUNE 14, 2023

Scientists discover small RNA that regulates bacterial infection People with weakened immune systems are at constant risk of infection. Abell Chair in Molecular and Cellular Biology – and Pengbo Cao, a postdoctoral researcher in Whiteley’s lab, discovered a gene that drives the switch. and Nelson D.

RNA 40

RNA Regulation Scientist Bacteria 40

Scienmag

APRIL 21, 2021

Suppressing a gene regulator could reduce pancreatic cancer resistance to vital chemotherapeutic treatment. Targeting an RNA to interrupt its activity could improve patient response to therapy and increase their overall survival. […].

RNA 70

RNA Gene Regulation Research 70

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.

Gene Silencing 66

Gene Silencing Gene RNA Contract Manufacturing 66

XTalks

JANUARY 21, 2025

The tricuspid valve, one of the heart’s four valves, regulates blood flow from the right atrium to the right ventricle, preventing backflow between these chambers. According to Geneoscopy , ColoSense is the first non-invasive colorectal cancer screening test to provide a dynamic view of disease activity by using RNA biomarkers.

FDA Approval 105

FDA Approval Manufacturing Development RNA 105

BioTech 365

FEBRUARY 25, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Super-resolution RNA imaging in live cells.Ribonucleic acid (RNA) is key to various fundamental biological processes. It transfers genetic information, translates it into proteins or supports gene regulation.

RNA 40

RNA Genetics Protein Gene 40

Roots Analysis

AUGUST 31, 2023

Overview of Gene Switch The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E. Gene switches are sites on genes where regulatory molecules can bind to trigger transcription process, leading to expression of a particular gene.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

Delveinsight

SEPTEMBER 23, 2021

858 Therapeutics closes USD 60 Million series A round to drug RNA modulation. Now, they are ready for their next venture, which is launching RNA biotech 858 Therapeutics with a USD 60 million Series A fundraising. 858’s drug candidates include proteins, which regulate RNA and immune response. Jeffrey Stafford, Ph.D.,

RNA 61

RNA Gene Therapy Gene Protein 61

Roots Analysis

OCTOBER 22, 2023

RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. Non-coding RNAs include antisense oligonucleotides (ASOs) and RNA aptamers.

RNA 40

RNA Protein Genetics Gene Expression 40

The Pharma Data

MARCH 7, 2022

By combining CRISPR technology with a protein designed with artificial intelligence (AI), it is possible to awaken individual dormant genes by disabling the chemical “off switches” that silence them. The chemical modifications that regulate gene activity are called epigenetic markers. it can be reawakened.

Protein 52

Protein Gene Genome Genomics 52

Roots Analysis

OCTOBER 30, 2023

In the past few years, Next Generation RNA therapeutics have emerged as one of the key therapeutic modalities in the modern healthcare industry. These RNA based therapeutics play a crucial role in protein production and regulation of gene functions.

RNA 40

RNA Vaccine Vaccination Development 40

Delveinsight

JULY 24, 2021

In addition to this, Angelman syndrome pipeline also involves gene therapy candidates such as GTX-101 which is a therapy of GeneTx Biotherapeutics. Another therapy by the same company named GTX-102 is also an antisense therapy; however, it silences gene expression by binding to the RNA molecule that regulates its expression.

Gene Therapy 96

Gene Therapy Gene Gene Expression RNA 96

Scienmag

DECEMBER 22, 2020

Credit: Kung-Chi Chang/Spector lab, 2020 Cold Spring Harbor Laboratory (CSHL) scientists have discovered a gene-regulating snippet of RNA that may contribute to the spread of many breast cancers. The same strategy, […].

RNA 52

RNA Gene Scientist Regulation 52

XTalks

MAY 4, 2021

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. It’s a great tool for controlling gene expression.”. pyogenes dCas9.

DNA 98

DNA Gene Gene Silencing Genome 98

The Pharma Data

JANUARY 19, 2021

A naturally occurring system for tuning CRISPR-Cas9 expressing in bacteria, identified in a study published in Cell , could have implications for gene editing therapies as well. A CRISPR-Cas9 system has two components: the Cas9 guide RNA that directs the system to edit a particular gene, and the CRISPR “scissors” that make the cut.

Bacteria 52

Bacteria Gene Editing Research RNA 52

Pharmaceutical Technology

DECEMBER 9, 2022

The move is particularly timely given the significant growth expected in global sales of cell and gene therapies. Ribonucleic acid (RNA) based technologies are among many innovative approaches explored to tackle the Covid-19 pandemic. However, messenger RNA (mRNA) based vaccines present particular analytical challenges.

Development 130

Development Production Manufacturing Vaccine 130

pharmaphorum

DECEMBER 14, 2022

The two partners have revealed the first clinical data from a phase 2b trial of a personalised vaccine consisting of 34 mRNAs, each targeting mutations – identified by gene sequencing – that are thought to be driving a patient’s cancer. They also said they plan to discuss the phase 2b results with regulators.

Vaccine 98

Vaccine Vaccination Trials Gene Sequencing 98

Pharmaceutical Technology

JUNE 4, 2023

Innovation S-curve for the pharmaceutical industry Transcription factors for genetically modified cells is a key innovation area in the pharmaceutical industry Transcription factors are proteins which control gene expression by controlling the rate of transcription of genetic information from DNA to RNA.

Genetics 130

Genetics Gene Expression DNA Antibody 130

pharmaphorum

MARCH 9, 2022

The XTEND-1 trial also showed that BIVV001 was better at reducing the annualised rate of bleeding episodes than patients’ previous therapies, according to Sanofi and Sobi, which said they will now talk to regulators to chart a path forward to approval.

Protein 98

Protein Gene Therapy Sales Regulation 98

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.

Gene Silencing 52

Gene Silencing RNA Protein Gene 52

pharmaphorum

MARCH 4, 2021

But with Novartis failing to convince European regulators about the merits of Xiidra last year, and Allergan’s ageing Restasis potentially facing generic competition, there’s a lot to play for and two biotechs are aiming to disrupt the market. Novartis paid Takeda $3.4

Gene Silencing 98

Gene Silencing FDA Approval Trials Marketing 98

XTalks

APRIL 6, 2022

XTALKS WEBINAR: Using CRISPR interference (CRISPRi) Viability Screens to Map Long Noncoding RNA Dependencies in Tumor Cells. Register for this webinar to get an overview of the role of long non-protein coding RNAs (lncRNAs) in gene regulations. Live and On-Demand: Thursday, April 21, 2022, at 11am EDT (4pm BST/UK).

DNA 105

DNA In-Vitro Doctor Doctors 105

pharmaphorum

SEPTEMBER 21, 2020

The drug works by reducing the production of ApoC-III, a protein that regulates plasma triglycerides. Akcea – an affiliate of Ionis that is set to become a wholly-owned subsidiary shortly – specialises in antisense drugs that target RNA to prevent the expression of errant genes that cause diseases.

Drugs 80

Drugs RNA Medicine Genetics 80

XTalks

MAY 4, 2021

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. These modifications regulate gene expression without altering the sequence or structure of DNA.

DNA 52

DNA Gene Gene Silencing 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. Oxlumo (lumasiran) was also approved in the European Union last week, making it the third from Alnylam’s pipeline of RNA interference therapeutics to make it to market.

FDA Approval 52

FDA Approval Gene Silencing Drugs RNA 52

The Pharma Data

MARCH 3, 2022

This finding stemmed from more than two decades of her basic research into a gene in pancreatic islets called TXNIP. The current study extends on that finding and provides crucial mechanistic and clinical insights into the beneficial effects of verapamil in Type 1 diabetes, using proteomics analysis and RNA sequencing.

Insulin 52

Insulin Gene Expression Gene Protein 52

Delveinsight

JANUARY 18, 2021

Angelman syndrome is a result of the missing or dysfunctional gene known as UBE3A. The gene is responsible for the normal and healthy development of an individual physically as well intellectually. Thus, restoring the presence of this particular gene can help to restore several of the functions of the brain.

Marketing 52

Marketing Gene Drugs Protein 52

The Pharma Data

SEPTEMBER 22, 2020

Novartis Gene Therapies to initiate new pivotal confirmatory study to evaluate use of AVXS-101 intrathecal (I T ) formulation in older patients with SMA to further support registration. Novartis Gene Therapies remains confident in the overall benefit-risk profile for patients on treatment.

Clinical Development 52

Clinical Development Gene Therapy Development Gene 52

FDA Law Blog

JANUARY 12, 2025

Since that time, it was formalized in FDA regulations (21 CFR 314 Subpart H) in 1992, codified in the Food, Drug, & Cosmetic Act by FDAMA (21 USC 356(c)) in 1997, revised by FDASIA in 2012, and described in guidance, most importantly, in the 2014 Expedited Programs for Serious Conditions Drugs and Biologics (2014 Guidance).

Clinical Trials 64

Clinical Trials Clinical Trials Trials Drugs 64

XTalks

AUGUST 12, 2020

Studies have identified the presence of two types of HIV-infected CD4+ T cells: ‘Transcriptionally inactive’ cells that do not typically produce viral RNA or viral proteins. Transcriptionally active’ cells in which HIV RNA is actively transcribed to make copies of the virus (despite long-term ART).

RNA 52

RNA Protein DNA In-Vivo 52

pharmaphorum

JULY 21, 2021

Novartis has revealed that the FDA has accepted its resubmission for cholesterol-lowering drug Leqvio – rejected unexpectedly by the US regulator last year – and has set a new action date of 1 January 2022. Meanwhile, sales of gene therapy Zolgensma for spinal muscular atrophy shot up more than 50% to $315 million.

Sales 52

Sales Gene Therapy RNA Production 52

XTalks

NOVEMBER 7, 2022

Such information can reveal details about tumor staging, tumor progression, heterogeneity, gene mutations and clonal evolution. The first option is to design a personalized small NGS gene panel based on the somatic mutations that were detected in the patient, and this can be used for monitoring disease progression.

Clinical Trials 98

Clinical Trials Clinical Trials Trials FDA Approval 98

XTalks

AUGUST 30, 2022

We know that a lot of different pathologies, especially as we’ve entered the era of molecular medicine and gene therapies, cause many of the same symptoms and work through final common pathways. Such therapies may involve small interfering RNA (siRNA) or oligonucleotides in viral transfection platforms, large molecules and antibodies.

Trials 83

Trials Drug Delivery Drugs Gene 83

The Pharma Data

MARCH 5, 2021

This will be adapted to the needs of messenger RNA vaccine production for CureVac’s CVnCoV, the manufacturing of which is highly complex. Production will take place in a new high-tech production facility that was already under construction at the Novartis Kundl, Austria site.

Manufacturing 52

Manufacturing Vaccination Vaccine Production 52