pharmaphorum

JANUARY 29, 2021

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. The exact mechanism depends on the disease in question.

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Gene Editing Gene Gene Therapy In-Vivo 145

pharmaphorum

JANUARY 10, 2023

Astellas has licensed rights to a drug developed by Selecta Biosciences that could make more patients eligible for treatment with its gene therapy for inherited neuromuscular disorder Pompe disease. The post Astellas licenses companion drug for Pompe gene therapy appeared first on.

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Gene Therapy Gene Licensing Licensing 119

Pharmaceutical Technology

AUGUST 11, 2022

Alnylam Pharmaceuticals and collaborators have identified rare mutations in the INHBE gene that is expressed in the liver, related to a lower waist-to-hip ratio for body mass index (BMI), which is often used as an indicator of abdominal fat and is correlated with the risk of type 2 diabetes (T2D) and coronary heart disease.

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Gene Biobanking Gene Expression In-Vitro 130

XTalks

AUGUST 5, 2020

Single cell RNA sequencing technology has been extensively applied to understand heterogeneity among tumor cells, within the tumor microenvironment and during cell development. The barcode sequence is followed by a unique molecular identifier, which enables accurate digital counting of gene transcripts.

Gene Expression 112

Gene Expression Development RNA Drugs 112

Pharmaceutical Technology

JANUARY 24, 2023

The investigational adeno-associated virus (AAV) gene therapy candidate NGN-401 is claimed to be the first to deliver the full-length human MECP2 gene using the company’s Expression Attenuation via Construct Tuning (EXACT) gene regulation technology.

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Gene Therapy Gene Gene Expression Regulation 208

Drug Discovery Today

APRIL 24, 2023

Hussman Institute for Human Genomics at the University of Miami Miller School of Medicine have found that variations in chromatin accessibility, and thus gene expression, may explain why people of European descent with APOE4 gene variants have a greater risk of developing Alzheimer’s disease than people of African descent with similar genetics.

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Gene Gene Expression Genomics Genome 80

Scienmag

SEPTEMBER 30, 2021

Rhizobium-legume symbioses lead to the development of new plant organs on the roots […]. Crop legumes are an integral part of sustainable agriculture, as several of these species represent an important protein source for both human and animal populations.

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Gene Expression Bacteria Gene Protein 80

Scienmag

JULY 6, 2022

A University of Minnesota Twin Cities-led team has developed a new technique that allows scientists and engineers to, for the first time, visualize mRNA molecules in the brains of living mice.

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Gene Expression Research Gene Scientist 90

Scienmag

FEBRUARY 18, 2021

Mount Sinai researchers have discovered that Polycomb complexes, groups of proteins that maintain gene expression patterns, are essential for proper skin development, according to a paper published in Genes & Development on February 18.

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Gene Expression Development Research Gene 72

Camargo

JULY 8, 2021

Each month, Camargo’s “In the News” series highlights important changes and advancements in the regulatory and development space and explores how those changes could impact your program. The report is a useful read to keep current on a topic of interest at the FDA and in drug development as a whole.

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Development Bioequivalency Gene Expression Drugs 130

Scienmag

MAY 16, 2022

Just as it’s hard to understand a conversation without knowing its context, it can be difficult for biologists to grasp the significance of gene expression without knowing a cell’s environment.

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Gene Expression Engineer Gene Development 97

Scienmag

DECEMBER 22, 2020

Blocking gene expression in mitochondria in mice stops cancer cells from growing Credit: Hauke S. Hillen A newly developed compound starves cancer cells by attacking their “power plants” – the so-called mitochondria. The new compound prevents the genetic information within mitochondria from being read.

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Gene Expression Genetics Gene Drugs 95

BioSpace

JULY 11, 2022

Epic Bio aims to transform genetic medicine by developing a new class of drugs that target the epigenome to alter gene expression.

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Genetics Gene Expression Medicine Engineer 81

BioTech 365

DECEMBER 1, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Researchers identify key RNA “gatekeeper” in gene expression, pointing to possible new drug targets.An

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Gene Expression RNA Gene Research 40

Scienmag

DECEMBER 28, 2021

Philadelphia, December 28, 2021 – In the central nervous system, microglial cells play critical roles in development, aging, brain homeostasis, and pathology. Recent studies have shown variation in the gene-expression profile and phenotype of microglia across brain regions and between different age and disease states.

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Gene Expression Gene Development 85

Scienmag

DECEMBER 10, 2020

Credit: Nucleic Acids Research Scientists at Tokyo Institute of Technology decipher how to quantitatively assess the effects of specific epigenetic changes on the rate of transcription by developing a mathematical model. For this, they successfully generated reconstituted chromatin bearing histone modifications in vitro.

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Gene Expression In-Vitro Gene Scientist 52

XTalks

FEBRUARY 21, 2025

Related: Obesity-Focused Metsera Launches IPO Amid Much Anticipation Aardvarks approach centers on developing novel, small-molecule drugs that harness the bodys natural hunger-regulating mechanisms. Alongside ARD-101, Aardvark is developing ARD-201, a fixed-dose combination of ARD-101 with a dipeptidyl peptidase-4 inhibitor.

Genetics 59

Genetics Hormones Gene Expression Gene 59

pharmaphorum

NOVEMBER 12, 2021

Researchers in the US have developed an artificial intelligence-based tool that is able to predict COVID-19 symptoms and suggest which FDA-approved drugs might be used to treat patients. It could also be made more powerful by incorporating personal genetic information and gene expression profiles in tissues such as the lungs, they suggest.

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Doctors Doctor Drugs Gene Expression 135

Medical Xpress

FEBRUARY 15, 2023

Traffic noise may play an essential role in the development and deterioration of ischemic heart disease.

Gene Expression 69

Gene Expression Cardiology Gene Research 69

pharmaphorum

MARCH 10, 2021

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. The post bluebird seeks gene therapy trial restart after cancer scare appeared first on.

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Gene Therapy Gene Trials Gene Expression 59

BioTech 365

OCTOBER 6, 2020

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: The plant hormone auxin may promote disease by regulating virulence gene expression.Scientists have long known that the plant hormone auxin controls many aspects of plant growth, development, … Continue reading →

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Hormones Gene Expression Regulation Gene 40

Scienmag

MARCH 3, 2021

Using the model organism Caenorhabditis elegans, researchers at the University of Cologne have developed an ‘aging clock’ that reads the biological age of an organism directly from its gene expression, the transcriptome.

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Gene Expression Genomics Genome Gene 60

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.”.

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DNA Gene Gene Silencing Genomics 98

pharmaphorum

AUGUST 2, 2021

The agreement gives the company rights to spherical nucleic acid (SNA) drugs – nanoparticles with nucleic acids bound to their surfaces – which can be used to get oligonucleotides into cells to modify gene expression efficiently, and without the toxicity caused by some other delivery mechanisms.

Gene Expression 105

Gene Expression Gene Licensing Licensing 105

pharmaphorum

JUNE 27, 2022

The safety of Astellas’ gene therapy portfolio has been thrust into the spotlight once again, after the FDA placed a clinical hold on a trial of its Pompe disease candidate AT845. The delivery vector – an AAV8 capsid – is designed to allow gene expression directly in target tissues like skeletal muscle.

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Gene Therapy Gene Gene Expression Trials 52

XTalks

MARCH 26, 2025

Here are five key examples of current research and innovation driving the field forward between 2024 and early 2025: Third-Generation Antiseizure Medications Show Promise Recent clinical data shared in Epilepsia spotlighted Xcopri (cenobamate), developed by SK Life Science.

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Life Science Gene Expression Trials Drugs 66

Pharmaceutical Technology

JUNE 13, 2023

The aberrant protein complex is essential for regulating leukaemia-promoting gene expression. A resistant mutation, MEN1-M3271, developed in one of 29 patients, detected at the end of Cycle 4, but the patient maintained stable disease through Cycle 7. The median age of RP2D patients in the study was 70.5 years (22–86 years).

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Gene Expression Protein Sales Genetics 130

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

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.

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Gene Gene Therapy Gene Expression Regulation 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.

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Gene Therapy Gene Gene Expression RNA 96

BioTech 365

MARCH 24, 2021

Ocean Genomics Partners With Geninus to Co-Develop RNA-Based Biomarkers and Advance Research Ocean Genomics Partners With Geninus to Co-Develop RNA-Based Biomarkers and Advance Research PITTSBURGH–(BUSINESS WIRE)–#RNA–Ocean Genomics (Pittsburgh, PA), a world leader in AI-based gene expression analysis and RNA biomarker … Continue (..)

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RNA Genomics Genome Gene Expression 40

Scienmag

MAY 23, 2021

Press release – Abstract 1394: Alterations in clock genes expression in Eutopic and Ectopic Endometrial Tissue New research suggests that night shift work is linked to menstrual irregularity and increased chance of developing endometriosis According to a study being presented at the 23rd European Congress of Endocrinology (e-ECE 2021), on Sunday (..)

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Gene Expression Development Research Gene 41

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.

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Protein Gene Genomics Genome 52

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. Which means it’s not any show, it’s the show,” he said.

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Regulation Genetics Scientist RNA 52

The Pharma Data

JULY 11, 2021

Eli Lilly and Company (NYSE: LLY) announced new Phase 2 data showing that gene expression changes induced by mirikizumab in patients with ulcerative colitis (UC) over a 12-week induction treatment were maintained for up to one year. For methodology, see the “About the Studies” section below.

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Regulation Gene Gene Expression Packaging 52

XTalks

MARCH 13, 2024

Biomarker testing is being pursued to identify biological signs of the disease and provide an approach to develop personalized treatment plans for each patient. As this technology continues to evolve, it is expected to help identify glaucoma in its nascent stages of development.

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Gene Therapy Gene Life Science DNA 111

The Pharma Data

FEBRUARY 24, 2022

Researchers at the University of North Carolina at Chapel Hill and the UNC Lineberger Comprehensive Cancer Center have uncovered a new role of a chromatin-modulatory enzyme, termed EZH2, during cancer development. They then developed a new therapeutic approach with a potent small-molecule inhibitor of this enzyme.

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Scientist Gene Expression Gene Genomics 52