Drug Discovery World

JUNE 23, 2023

Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US. The Food and Drug Administration (FDA) has granted accelerated approval to Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus (AAV) based gene therapy for the treatment of DMD.

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Gene Therapy FDA Approval Gene Protein 97

Scienmag

OCTOBER 27, 2021

The rapid scientific advancements that followed the mapping of the human genome have revealed just how staggeringly complex the world of genetics is. We now know that proteins are not just the products of genes, but that they also interact with genes, influencing and regulating the rhythm of their expression.

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Gene Genome Genomics Genetics 72

Drug Discovery World

AUGUST 16, 2023

Decoding ‘junk DNA’ The Human Genome Project and subsequent studies discovered that most of our DNA (approximately 98%) does not actually code for proteins, with humans having approximately 20,000 tox 25,000 protein-coding genes.

RNA 52

RNA Genome Genomics DNA 52

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

JULY 14, 2022

Every gene in a cell is expressed at exact levels due to complex gene regulatory networks. For example, when T cells (a type of white blood cells that fight off infections and cancer) are activated in our immune systems, thousands of proteins in these cells change. Using CRISPR Technology to Disrupt Networks of Immune Genes.

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Gene Regulation Protein Gene Editing 98

Scienmag

DECEMBER 22, 2021

Genes code for proteins – the building blocks of life. The current definition of a gene only accounts for around 1.5%

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Genome Genomics DNA Gene 63

BioTech 365

DECEMBER 1, 2020

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Genomics, gene-editing and the Blue Revolution.Aquaculture—the farming of fish and shellfish, is the world’s fastest growing primary industry.

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Gene Editing Genome Genomics Gene 40

Drug Discovery World

NOVEMBER 1, 2022

Researchers say they have successfully used a cell’s natural process for making proteins to ‘slide’ genetic instructions into a cell and produce critical proteins missing from those cells. . Two methods currently used to deliver protein-making packages into cells, ‘mini promoters’ and serotype-mediated gene expression, vary widely.

Gene Expression 52

Gene Expression Gene Packaging Packaging 52

BioTech 365

OCTOBER 26, 2020

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: A ‘genomic rosetta stone’ for discovering the rules of gene regulation.As

Genome 40

Genome Genomics Gene Regulation 40

pharmaphorum

AUGUST 4, 2022

Unlocking the secrets of the human genome has long been an ambitious pursuit for researchers around the world. Today, the landscape of genomic testing and research is rapidly progressing, with significant scientific and technological advances driving a paradigm shift in the understanding of oncology at a molecular level.

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Genome Genomics Genome Project DNA 52

Medical Xpress

MAY 4, 2023

The p53 gene is one of the most important in the human genome: the only role of the p53 protein that this gene encodes is to sense when a tumor is forming and to kill it. While the gene was discovered more than four decades ago, researchers have so far been unsuccessful at determining exactly how it works.

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Scientist Gene Genome Genomics 105

BioTech 365

JUNE 4, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: The cut and restore protein trick: Self-excising designer proteins.Our proteome is much bigger than our genome because one gene produces several variants of proteins called protein isoforms, … Continue reading →

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Protein Genome Genomics Gene 40

Scienmag

NOVEMBER 20, 2021

Professor Norikazu Ichihashi and his colleagues at the University of Tokyo have successfully induced gene expression from a DNA, characteristic of all life, and evolution through continuous replication extracellularly using cell-free materials alone, such as nucleic acids and proteins for the first time.

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DNA Gene Expression Genome Genomics 96

Scienmag

MAY 11, 2021

MIT researchers have determined the virus’ protein-coding gene set and analyzed new mutations’ likelihood of helping the virus adapt. CAMBRIDGE, MA — In early 2020, a few months after the Covid-19 pandemic began, scientists were able to sequence the full genome of the virus that causes the infection, SARS-CoV-2.

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Genome Genomics Gene Protein 40

Medical Xpress

JANUARY 20, 2023

A team of researchers at the University of Texas Southwestern Medical Center has found that it is possible in mice to protect the heart from ischemia-reperfusion using the CRISPR-Cas9 gene editing system.

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Gene Editing Genome Genomics Gene 81

Drug Discovery World

AUGUST 21, 2023

Transcription factors are proteins that control every gene in a person’s genome by binding to DNA and flipping the on/off switches that govern gene expression. Talus Bio was also awarded a $2.0M At Talus Bio we believe ‘nothing is undruggable.’ The post $4.3

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Drugs Gene Expression Protein Gene 98

The Pharma Data

MARCH 15, 2022

The p53 protein protects our cells from cancer and is an interesting target for cancer treatments. Researchers at Karolinska Institutet in Sweden have now found an unusual way of stabilising the protein and making it more potent. The new protein also proved to be more stable than ordinary p53 and capable of killing cancer cells.

Protein 52

Protein Research Genetics DNA 52

BioTech 365

JUNE 16, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: How long-known genes continue to surprise researchers.The human genome was sequenced around 20 years ago.

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Gene Research Genome Genomics 40

Drug Discovery World

NOVEMBER 14, 2023

A Phase Ib trial has shown low-density lipoprotein cholesterol (LDL-C) reductions up to 55% and blood PCSK9 protein reductions up to 84% after a single infusion of Verve Therapeutics’ Verve-101. It is caused by a single gene mutation that impairs the body’s ability to mediate LDL cholesterol.

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In-Vivo Gene Editing Gene Genome 52

Drug Discovery World

OCTOBER 6, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

Drugs 52

Drugs Gene Editing Genome Genomics 52

Pharmaceutical Technology

FEBRUARY 15, 2023

Innovation S-curve for the pharmaceutical industry CRISPR nuclease is a key innovation area in pharmaceutical development CRISPR, which refers to clustered regularly interspaced short palindromic repeats, are bacteriophage-derived DNA sequences that had previously infected the prokaryote and are found in the genomes of bacteria and archaea.

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In-Vivo Genome Genomics Gene Editing 162

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. He added: “We have seen no significant gene misregulation attributable to the insertion event.

Gene Therapy 59

Gene Therapy Gene Trials Gene Expression 59

Scienmag

MAY 7, 2021

The two meters of -stretched- DNA contained in human cells are continuously twisting and untwisting to give access to genetic information: when a gene is expressed to generate a protein, the two strands of DNA are separated to give access to all the machinery necessary for this expression, resulting in an excessive accumulation of coiling […]. (..)

DNA 62

DNA Regulation Genome Genomics 62

Scienmag

FEBRUARY 21, 2021

‘Exploring one of the biggest challenges facing genomics today – understanding genetic variants’ SEATTLE (February 22, 2021) – Global experts performing leading edge research in precision medicine, functional genomics, protein science, and variant interpretation and prediction will share their expertise and insights April 5-7 (..)

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Genome Genomics Genetics Protein 40

The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., “Accessing state-of-the-art LNP technology through this collaboration will add momentum to our gene editing efforts for the benefit of patients.”

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Gene Therapy Gene Gene Editing In-Vivo 40

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. What are the advantages compared to other gene modulating approaches?

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Gene Expression Gene Genetic Disease RNA 57

XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

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In-Vivo Gene Editing Trials Gene 52

Bioengineer

JANUARY 16, 2024

This mutation hinders the drug’s binding to the mutant EGFR protein. Osimertinib, however, can covalently bind to the T790M and cysteine-797 (C797) residue at the protein’s ATP binding site, overcoming resistance mechanisms. In the same study, those with T790M loss had a shorter treatment duration (6.1 months vs. 15.2

Genome 119

Genome Genomics Protein In-Vitro 119

Scienmag

SEPTEMBER 16, 2020

Credit: Matt Jaremko/CSHL Joshua-Tor lab The most basic activity of a living thing is to turn one copy of its genome into two copies, crafting one cell into two. That replication event begins with a set of proteins–the Origin of Replication Complex (ORC).

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Genome Genomics Protein Gene 42

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. Epigenetics is still largely unexplored and referred to as the ‘dark matter’ of the genome.

Gene Expression 52

Gene Expression Gene DNA RNA 52

pharmaphorum

APRIL 4, 2022

The largest-ever study of genetics in Alzheimer’s disease patients has identified 42 new genes that appear to be linked to the neurodegenerative disorder. ” The study was carried out using the genomes of 100,000 people with Alzheimer’s and 600,000 healthy individuals.

Gene 52

Gene Genetics Research Development 52

WCG Clinical

NOVEMBER 17, 2023

Although the NIH Guidelines were originally written with non-clinical laboratory research in mind, they also apply to human gene transfer (HGT) research, wherein rsNA or rsNA-containing products are administered to research participants. Since then, however, certain genetic engineering technologies (e.g.,

Genome 52

Genome Genomics Gene Editing Genetic Engineering 52

Drug Discovery World

SEPTEMBER 27, 2023

Embarking on a new era of medicine The advent of genomics has ushered in the era of personalised medicine, enabling us to analyse the genetic makeup of individuals with unprecedented accuracy. HKG Epitherapeutics is a biotechnology company developing novel tools for the early detection of cancer and promoting healthy aging.

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DNA Genetics Gene Medicine 113

Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. million price point per dose.

Gene Therapy 52

Gene Therapy Gene Manufacturing Medicine 52

Drug Discovery World

NOVEMBER 27, 2023

The European Commission has extended use of the therapy to include children with CF aged two through five years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. “As

Protein 52

Protein Gene Medicine Gene Therapy 52

pharmaphorum

JULY 20, 2022

Armed with a $100 million second-round financing, CAMP4 Therapeutics is preparing to start the first clinical trial of a drug targeting regulatory RNA (regRNA) molecules that can be used to fine-tune the expression of genes. ” The post CAMP4 raises $100m to take lead RNA drugs into clinic appeared first on.

RNA 52

RNA Genetic Disease Drugs Protein 52

Scienmag

APRIL 15, 2021

Team of Japanese and European scientists identify a novel genetic mitochondrial disorder by analyzing DNA samples from three distinct families Credit: Fujita Health University DNA ligase proteins, which facilitate the formation of bonds between separate strands of DNA, play critical roles in the replication and maintenance of DNA.

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Genetics DNA Genome Genomics 105