Pharmaceutical Technology

JULY 29, 2022

However, in patients with urea cycle disorders, genetic defects result in inadequate amounts of the enzymes needed to convert nitrogen into urea. Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression. Typically, nitrogen in the blood is removed in the form of urea.

RNA 245

RNA Gene Expression Gene Gene Therapy 245

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: How easy was it to transfer theoretical research into a viable business?

Gene Expression 59

Gene Expression Gene Genetic Disease RNA 59

Drug Discovery World

AUGUST 16, 2023

A new study has shed light on the genetic messages encoded by genes within ‘triple negative’ breast cancers (TNBC), and shows they could predict response to chemotherapy. Although immune features remained relatively stable, RNA-based features related to DNA damage repair pathways changed significantly.

Genetics 52

Genetics Gene Hormones RNA 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. Genetic variations inherited from our parents shape our response to drugs, disease susceptibility, physiology and metabolism.

DNA 115

DNA Genetics Gene Medicine 115

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

JANUARY 29, 2021

Genome editing is an exciting but still nascent field, and companies in the area face as many obstacles as they do opportunities. Then over the next two or three years, gene therapy was accepted as something that companies got involved in, and several biotechs have been bought up by big pharma.”. The genomic medicine journey.

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Gene Editing Gene Gene Therapy In-Vivo 145

Scienmag

JANUARY 11, 2021

These biomarkers are epigenetic, meaning they involve changes to molecular factors that regulate genome activity such as gene expression independent of DNA sequence, and can be passed down to future generations. In a […].

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Gene Expression DNA Genome Genomics 41

XTalks

AUGUST 5, 2020

The detailed complexity of these processes can be captured by creating models that combine correlates of gene and protein expression, providing insight into the molecular composition of tissues. Visium Spatial Gene Expression Solution. Feature barcoding using gel beads. Leading Innovations in Single Cell Technology.

Gene Expression 112

Gene Expression Development RNA Drugs 112

Pharma Marketing Network

DECEMBER 21, 2020

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., 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.

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

Drug Discovery World

DECEMBER 12, 2022

Other research updates include a study by NIH 5 researchers using post-mortem brain tissues to understand genomic differences in individuals with attention deficit hyperactivity disorder. The study advances our understanding of ADHD by showing how the condition is tied to changes in how certain genes are expressed in the brain.

Drugs 52

Drugs Gene Editing Genome Genomics 52

Drug Discovery World

OCTOBER 4, 2023

Scientists have discovered the link between gene expression, DNA methylation, and brain structural changes in depression, revealing new potential therapeutic targets. Parallel findings in humans underscored the involvement of interferon-related genes in major depressive disorder.

Research 52

Research Gene DNA Gene Expression 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.

RNA 52

RNA Drugs Gene Expression Gene 52

pharmaphorum

MARCH 10, 2021

The US biotech said the suspected serious adverse reaction reported in the phase 1/2 study of LentiGlobin gene therapy for sickle cell disease was unlikely to be related to the virus vector used in the therapy, used to deliver the genetic material to the body. bluebird bio’s Philip Gregory.

Gene Therapy 59

Gene Therapy Gene Trials Gene Expression 59

Drug Discovery World

JULY 15, 2022

The first complete, gapless sequence of a human genome was published 1 April 2022 in a special issue of the journal Science 1. While The Human Genome Project mapped about 92% of the human genome two decades ago, sequencing the last 8% of the genome proved highly challenging.

Genome 52

Genome Genomics DNA Containment 52

The Pharma Data

JULY 11, 2021

Since then, there has been an increasing body of findings that the microbiome, which is sometimes also referred to as the second human genome, is not only of central importance for digestion, but also influences, if not controls, at least a large number of body functions. The immune system is mentioned particularly frequently.

Bacteria 52

Bacteria Genetics Regulation Scientist 52

Roots Analysis

AUGUST 31, 2023

Nutrigenomics is the science studying the relationship between human genome, nutrition and health. In part, the success of the Human Genome Project has also paved a path for the novel concept of nutrigenomics. Nutrigenomics tests are capable of determining the influence of genes on nutrient requirements and metabolism.

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Genome Genomics Genetics Gene 40

XTalks

FEBRUARY 11, 2021

The Human Genome Project recently marked 20 years since the publication of the first full sets of human genomic sequences, an endeavor that spanned well over a decade. Today, new next-generation sequencing technologies allow for the sequencing of complex genomes within just a day or two. Rosalind Franklin.

Genetics 119

Genetics DNA Genome Genomics 119

Drug Discovery World

AUGUST 1, 2023

opti-ox uses a dual genomic safe harbour approach to cell programming, and the granted US patent covers the use of opti-ox to deterministically activate genetic code in any eukaryotic (cell with a nucleus) cell type. bit.bio has secured a foundational US patent for opti-ox, its precision cell programming and manufacturing technology.

Gene Expression 52

Gene Expression Genome Genomics Genetics 52

Drug Discovery World

DECEMBER 15, 2022

Gene editing tools such as zinc finger nucleases, transcription activator-like effector nucleases (TALEN) and clustered regularly interspaced short palindromic repeat (CRISPR) nucleases have been heralded for their enormous potential to treat diseases and genetic disorders. Expand representation across global populations.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

Drug Discovery World

MARCH 27, 2024

Pooled CRISPR screens offer the ability to knock out or perturb many genes in a single experiment from several hundred to tens of thousands of targets in parallel. Genome-wide screens are becoming the standard for drug discovery to power unbiased identification of drug targets across gene regulatory networks.

Genome 52

Genome Genomics Gene Expression RNA 52

XTalks

MARCH 7, 2024

Breast Cancer Multiomics: Unified Insights in Tumor Heterogeneity This on-demand webinar introduces a cutting-edge single-cell multiomics method designed for garnering detailed genetic insights into breast cancer. The test accurately identified a range of genetic abnormalities, including trisomies and microdeletions.

Hormones 115

Hormones Genetics Genome Genomics 115

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.”.

DNA 98

DNA Gene Gene Silencing Genome 98

Drug Discovery World

APRIL 11, 2024

Pooled CRISPR screens offer the ability to knock out or perturb many genes in a single experiment from several hundred to tens of thousands of targets in parallel. Genome-wide screens are becoming the standard for drug discovery to power unbiased identification of drug targets across gene regulatory networks.

Genome 52

Genome Genomics Gene Expression RNA 52

Drug Discovery World

MARCH 27, 2024

Pooled CRISPR screens offer the ability to knock out or perturb many genes in a single experiment from several hundred to tens of thousands of targets in parallel. Genome-wide screens are becoming the standard for drug discovery to power unbiased identification of drug targets across gene regulatory networks.

Genome 52

Genome Genomics Gene Expression RNA 52

Drug Discovery World

JULY 18, 2023

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. It also enables the downregulation of gene expression by directly knocking out the cytoplasmic mRNA transcripts.

DNA 98

DNA Genome Genomics RNA 98

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

Drug Discovery World

APRIL 14, 2023

At the beginning of 2023, proteomics company Nautilus Biotechnology announced a partnership with the Translational Genomics Research Institute (TGen), to study the specific proteins at work in the rare and often fatal childhood cancer, diffuse intrinsic pontine glioma (DIPG). BMC Bioinformatics. 2013;14 Suppl 2(Suppl 2):S24. Epub 2013 Jan 21.

Protein 52

Protein Genome Genomics DNA 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. We have shown that mRNA holds an unprecedented therapeutic potential for incurable genetic diseases, in particular liver conditions. We believe it can now do the same for rare diseases.” “We

RNA 52

RNA Protein Vaccination Vaccine 52

Drug Discovery World

APRIL 21, 2023

SLAS Student Poster Award The SLAS Student Poster Award recognises innovative research by students, graduate students, post- doctoral associates and junior faculty (less than four years in first academic appointment) who are chosen to present a poster during SLAS Europe 2023.

Doctors 52

Doctors Doctor Genetics In-Vitro 52

The Pharma Data

NOVEMBER 8, 2020

DB-OTO, Decibel’s investigational gene therapy to restore hearing in children with congenital deafness due to a deficiency in the otoferlin gene, is in preclinical studies, and Decibel expects to initiate clinical testing in 2022. Chief Executive Officer of Decibel. “We

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Gene Therapy Gene Gene Expression Genome 40

Drug Discovery World

JANUARY 23, 2023

Increasing interest in CRISPR-Cas gene editing systems over recent years has delivered innovative developments in precision genome engineering technologies, holding enormous potential for applications across the healthcare sector. The success of base editing has led to rapid advances toward commercialisation and clinical application.

Gene Editing 52

Gene Editing Gene Engineer DNA 52

XTalks

SEPTEMBER 21, 2020

A study from the University of California San Francisco (UCSF) has revealed that women with Alzheimer’s do better than men with the disease due to a genetic advantage conferred by their extra X chromosome. Increasing KDM6A expression in male mice led them to be more resilient to the effects of amyloid beta plaques. KDM6A Variant.

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Gene Protein Gene Expression Scientist 89

Drug Discovery World

FEBRUARY 6, 2023

A case in point is the emerging omics and other high-throughput laboratory technologies that allow simultaneous profiling of genetic alterations in combination with profiling thousands of molecules in plasma, cell populations, or single cells. This could be expanded to assessing predictors at baseline.

Big Data 52

Big Data RNA Bioinformatics Vaccination 52

Drug Discovery World

NOVEMBER 15, 2022

First off, genetically encoding desired features into living cells requires designing an appropriate vector, successfully transfecting it across the cell membrane, integrating the new DNA into the host’s genome, and finally, a reporting marker to examine changes. This approach has been around for a while.

DNA 98

DNA Drugs Antibody Engineer 98

XTalks

DECEMBER 24, 2020

From isolating SARS-CoV-2 in early January to sequencing its genome shortly thereafter and having a prototype vaccine against it within days, scientific process and progress have held steadfast throughout the pandemic. CRISPR Gene Editing Inventors Win Nobel Prize. The illness was later named coronavirus disease 2019 (COVID-19).

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Life Science Vaccination Vaccine Gene 111