Pharmaceutical Technology

JANUARY 19, 2023

AOC 1020 has been designed for the treatment of the underlying cause of FSHD, which is caused by the abnormal expression of a gene known as double homeobox 4 or DUX4. Avidity Biosciences’ AOC 1020 is intended to reduce the DUX4 mRNA and DUX4 protein expression in muscles in these patients. .

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Gene Expression Gene Protein RNA 246

Drug Discovery World

APRIL 17, 2024

DDW’s Diana Spencer speaks to Josh Mandel-Brehm , CEO of Massachusetts-based biotech CAMP4, to understand the role regulatory RNAs play in gene expression and how the founders overcame the challenge of launching a business based on brand new science. DS: You describe your therapeutics as RNA Amplifiers. How do they work?

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Gene Expression Gene Genetic Disease RNA 59

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. aeruginosa infections.

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RNA Regulation Scientist Bacteria 40

Scienmag

MAY 27, 2021

Capturing how RNA polymerase enzymes kick off transcription On scales too small for our eyes to see, the business of life happens through the making of proteins, which impart to our cells both structure and function.

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Gene Expression RNA DNA Protein 41

Drug Discovery World

MARCH 2, 2023

A new paper has solved the 20-year mystery of how epigenetic modifications act as traffic lights to control gene expression and could ultimately speed up the development of a new class of epigenetic cancer drugs. When it gives the green light, H3K4me3 allows RNA polymerase II to move along DNA, transcribing it into RNA as it moves.

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Gene Expression Gene DNA RNA 52

Scienmag

MARCH 17, 2021

An international team of scientists from the University of Turku, Finland and PennState University, USA have solved a long-standing mystery of how living organisms distinguish RNA and DNA building blocks during gene expression paving the way for the design of new antiviral drugs. All cellular […].

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Gene Expression RNA DNA Drugs 74

XTalks

AUGUST 5, 2020

In recent webinars by Genuity Science, formerly known as WuXi NextCODE, experts from the biotech and pharma industries spoke about leveraging the power of single cell RNA sequencing platforms and solutions in conjunction with machine learning technologies such as AI in cell biology and disease research. The Power of Single Cell Technology.

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Gene Expression Development RNA Drugs 112

Drug Discovery World

APRIL 17, 2024

ScaleBio’s technology enables high-throughput, instrument-free, cost-effective approaches to single-cell omics, and enables highly sensitive capture of guide RNAs matched with gene expression with their CRISPR Guide Enrichment Kit.

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Genome Genomics Gene Expression RNA 52

Drug Discovery World

APRIL 3, 2024

ScaleBio’s technology enables high-throughput, instrument-free, cost-effective approaches to single-cell omics, and enables highly sensitive capture of guide RNAs matched with gene expression with their CRISPR Guide Enrichment Kit.

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Genome Genomics Gene Expression RNA 52

Drug Discovery World

JULY 27, 2023

Use of RNA sequencing Researchers used experimental models to investigate the persistent mechanical allodynia that is associated with damaged sciatic nerve, comparing the therapeutic profiles of tianeptine to that of the antidepressant desipramine.

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RNA Gene Expression Gene Medicine 52

Drug Discovery World

FEBRUARY 13, 2023

SB: Transcriptomics is the study of genomic RNA transcripts in a biological system. RNA transcript expression levels can provide insight into underlying biology – eg. which genes are upregulated, and which ones are downregulated – which is very useful when studying different disease states. SB: Certainly.

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RNA Drugs Gene Expression Gene 52

Drug Discovery World

JUNE 25, 2024

The role of gene Gprc5a Professor Tadayoshi Hayata and Chisato Sampei, from Tokyo University of Science, along with their colleagues, conducted a series of experiments to identify druggable target genes downstream of PTH signalling in osteoblasts. The researchers treated cultured mouse osteoblast cells and mice with teriparatide.

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Gene Drugs Hormones Gene Expression 59

Camargo

JULY 27, 2021

Administration routes for gene therapies can differ depending on the indication target’s delivery needs. For example, it is beneficial to administer RNA treatments for some lung diseases directly to the disease site through inhalation. Gene Expression Considerations. Route of Administration Considerations.

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Gene Therapy Gene Gene Editing Genetics 238

Drug Discovery World

MAY 16, 2023

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to SiSaf’s small interfering RNA (siRNA) therapeutic for skeletal disorder Autosomal Dominant Osteopetrosis Type 2 (ADO2). SIS-101-ADO combines an siRNA that suppresses the expression of CLCN7 with Bio-Courier delivery technology.

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Gene Expression RNA Clinical Trials Clinical Trials 52

XTalks

JUNE 18, 2024

These platforms — Bobcat mRNATM, controllable self-replicating RNA (c-srRNA) and ZSCAN4 delivered by an RNA virus — represent the forefront of genetic therapy, harnessing the power of mRNA to combat diseases at their genetic roots. The essence of the c-srRNA platform lies in its innovative use of temperature as a regulatory mechanism.

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Protein RNA Genetics Clinical Trials 52

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.

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Genetics Gene Expression DNA Antibody 130

Drug Discovery World

JULY 8, 2024

Beard Distinguished Professor of Experimental Surgery and the Founding Director of the Translational Research Institute at Duke University, explores the rise of RNA therapeutics. However, the RNA landscape has drastically changed in recent years and we now know that the molecule is more than just a transient, linear carrier of information.

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RNA Protein Gene Expression Vaccination 59

Drug Discovery World

MARCH 21, 2023

In July 2022, DDW reported that molecular genetics company Mainz Biomed had enrolled its first patient in an international clinical study assessing the potential combination of gene expression (mRNA) biomarkers with a diagnostic test for colorectal cancer.

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Drugs Gene Expression RNA Clinical Trials 52

Drug Discovery World

AUGUST 16, 2023

Although immune features remained relatively stable, RNA-based features related to DNA damage repair pathways changed significantly.

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Genetics Gene Hormones RNA 52

pharmaphorum

MARCH 11, 2021

Join the Epigenetic Therapeutic Targets Summit – the only virtual summit dedicated to the clinical translation and optimization of therapeutic candidates targeting epigenetic mechanisms of gene expression.

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Gene Expression DNA Gene RNA 52

Drug Discovery World

FEBRUARY 14, 2024

A class of molecules playing a crucial role in the regulation of gene expression and other cellular processes can restore normal skin structure rather than producing a scar, a University of Manchester-led study suggests.

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Gene Expression Regulation Protein Gene 52

pharmaphorum

MAY 26, 2022

Gümrükcü is a specialist in gene expression profiling in stem cells, and has been working on ways to deliver stem cell transplants to treat cancer without the need for high-dose induction chemotherapy to destroy the patient’s bone marrow.

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Gene Expression RNA Licensing Licensing 95

Delveinsight

JULY 24, 2021

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. It is designed to target and inhibit the molecule that silences the paternal copy of the gene, called UBE3A antisense transcript (UBE3A-AS).

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Gene Therapy Gene Expression Gene RNA 96

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.

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Trials Gene Silencing RNA Gene Expression 59

The Pharma Data

MARCH 3, 2022

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.

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Insulin Gene Expression Gene Protein 52

The Pharma Data

MARCH 7, 2022

Cas9 is the protein used in the gene editing process called CRISPR. Cas9 binds and uses RNA as an address-tag. The system allows scientists, by synthesizing a specific “address-tag” RNA, to bring Cas9 to a precise location in genome and therefore cut and splice genes at specific sites.

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Protein Gene Genome Genomics 52

Scienmag

DECEMBER 1, 2021

BOSTON – An important player in the healthy development of female embryos turns out also to play a key role in regulating the behavior of chromosome loops and gene expression in both sexes, according to a new study by researchers at Massachusetts General Hospital (MGH).

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Gene Expression Gene RNA Research 61

Drug Discovery World

SEPTEMBER 27, 2023

Epigenetics, often referred to as the ‘software’ of gene regulation, encompasses chemical modifications to DNA, such as methylation, and modifications of other proteins that organise DNA in the nucleus, known as histones, as well as RNA.

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DNA Genetics Gene Medicine 115

Drug Discovery World

APRIL 16, 2023

Additional analyses are ongoing to identify biomarkers potentially associated with better outcomes, including gene expression profiles and PD-L1 expression. The association between this treatment approach and TMB will be further explored in upcoming studies.

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Vaccination Vaccine Immune Response Gene Expression 52

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.

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Drug Discovery World

AUGUST 3, 2022

Oligonucleotides act on the RNA level through different molecular pathways, but there is a major drawback when using them as drugs: their poor bioavailability and cellular uptake. This method is generally used to deliver small interfering RNA (siRNA), but it can also be used for larger oligonucleotides too. cells, such as diabetes.

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Bioavailability In-Vivo RNA In-Vitro 52

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).

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RNA Protein DNA In-Vivo 52

Drug Discovery World

APRIL 30, 2024

Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. mRNA is a molecule made up of a single strand of ribonucleic acid (RNA). Today, there are several other categories of RNA therapeutics with drugs already approved or currently in development.

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RNA Protein Vaccination Vaccine 52

Pharma Marketing Network

DECEMBER 21, 2020

those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies. greater safety, transient and reversible effect on gene expression).

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Genetics Medicine Gene Therapy Marketing 52

Roots Analysis

AUGUST 31, 2023

Mechanism of Gene Switch During the transcription process, the promoter region, which is located near the upstream end of each gene, binds to transcription factor, which is a specific type of protein. An additional level of genetic control is provided by gene switches that are located upstream of the promoter region.

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Gene Gene Therapy Gene Expression Regulation 40

Drug Discovery World

AUGUST 1, 2022

This technology defines gene expression patterns from rare cells harbouring latent HIV by generating millions of single-cell reaction containers in the form of water-in-oil emulsions, in which messenger RNA capture and virus DNA detection can be performed sequentially while maintaining segregation among cells.

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Gene Expression Scientist Gene Allergies 52

Drug Discovery World

FEBRUARY 6, 2023

This approach has led to the development of two software packages for RNA sequencing analysis and reproducible ribosomal profiling 1,2. For example, we recently collaborated with two sequencing laboratories to determine optimal parameters for RNA-Sequencing (RNA-Seq) in the context of a vaccine clinical trial. F1000Research.

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Big Data RNA Bioinformatics Vaccination 52