XTalks

SEPTEMBER 30, 2020

The research shows that the activity of the immune messenger type 1 interferon (IFN) protein is diminished, either by genetic mutations or an autoimmune attack by neutralizing antibodies against it, in a subset of COVID-19 patients. Related: Low T Cell Counts Observed in COVID-19 Patients. The analysis revealed that 23 (3.5

Antibody 135

Antibody Genetics Research In-Vitro 135

Drug Discovery World

APRIL 17, 2024

JMB: Regulatory RNAs (or regRNAS) are molecules that directly control the expression of nearby protein-coding genes. Recently, research published by Dr Richard (Rick) Young (Whitehead Institute) describes the key mechanism of how these regRNAs regulate gene expression. What are the challenges of drug discovery in this area?

Gene Expression 59

Gene Expression Gene Genetic Disease RNA 59

XTalks

SEPTEMBER 2, 2021

The AlzoSure Predict prognostic biomarker Alzheimer’s blood test detects levels of an unfolded conformational variant of the p53 protein in the blood using a proprietary antibody called U-p53AZ. It is known as the “guardian of the genome” given its central role in the DNA damage response. p53 in Alzheimer’s.

Protein 105

Protein DNA Antibody Genome 105

Drug Discovery World

APRIL 5, 2023

It targets a pathway that cancer cells are highly dependent upon, involving the heat shock factor 1 protein (HSF1). Mechanism of action HSF1 is a transcription factor, meaning that it controls the production of other important proteins in the cell, through its regulation of the process of reading the genetic code stored in DNA.

Drugs 52

Drugs Scientist Clinical Trials Clinical Trials 52

Roots Analysis

JANUARY 14, 2024

Like all drugs, biologics are regulated by the FDA. Biologics activate certain proteins or cells in your immune system to create specific responses to targets, while many conventional systemic drugs activate the entire immune system in a more generalized manner. The most widely used bacterial host system is Escherichia coli (E.

Contract Manufacturing 40

Contract Manufacturing Manufacturing Protein Bacteria 40

Drug Discovery World

JANUARY 23, 2023

Armed with this understanding, the researchers further demonstrated that the genetic inhibition of DGAT1 suppresses radioresistance. In addition, they discovered that cladribine, a clinical drug, activates DGKB and inhibits DGAT1. This action sensitised GBM cells to radiotherapy both in vitro and in vivo (in mouse models).

FDA Approval 52

FDA Approval Drugs In-Vivo In-Vitro 52

XTalks

APRIL 6, 2022

Cancerous cells and tumors release small fragments of DNA into the bloodstream called circulating tumour DNA (ctDNA). The researchers at the Cancer Research UK Cambridge Institute published this data from the LUng cancer CIrculating tumour DNA (LUCID) study in the journal Annals of Oncology.

DNA 105

DNA In-Vitro Doctors Doctor 105

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 genomic medicine journey. Pragmatic genomics.

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

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. Shares in bluebird ticked up following the announcement.

Gene Therapy 59

Gene Therapy Gene Trials Gene Expression 59

XTalks

MARCH 13, 2024

There are also studies examining gene therapy-based techniques to repair or replace genetic defects contributing to this disease. Secondly, other funded research is exploring differential gene expression regulated by DNA methylation. The Glaucoma Foundation has funded studies focusing on two specific areas within gene therapy.

Gene Therapy 111

Gene Therapy Gene Life Science DNA 111

Drug Discovery World

SEPTEMBER 5, 2022

Tumours (yellow) pervade the lungs of mice genetically prone to small cell lung cancer (left) on this computed tomography scan. The team discovered that RNF113A is regulated by a protein called SMYD3 that is highly expressed in small cell lung cancer and some other cancers.

Protein 52

Protein Drugs Medicine Research 52

Roots Analysis

OCTOBER 16, 2023

DNA cloning refers to the process of generating multiple copies of a particular DNA segment. DNA and Gene Cloning involves the isolation of a DNA sequence of any species ( often a gene ) and its insertion into a vector to enable growth without any alteration in the original DNA sequence.

DNA 40

DNA Gene Protein In-Vitro 40

pharmaphorum

SEPTEMBER 6, 2021

The biotech said that the US regulator ordered the pause on the study after liver tumours were seen in mice given the therapy in preclinical testing. PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down phenylalanine, an amino acid that is commonly found in many foods.

Gene Therapy 94

Gene Therapy Gene Genetic Disease DNA 94

Drug Discovery World

JULY 5, 2023

For example, in CAR-T-cell therapy, scientists can take T-cells from a patient and ex vivo (outside the body) genetically engineer them to effectively target and kill cancerous cells, then infuse them back in to the patient. Of course, if a patient’s tumour does not have this protein, then this type of treatment won’t be effective.

Research 98

Research Protein Scientist Immune Response 98

pharmaphorum

DECEMBER 21, 2020

But the tragic events of 2020 have meant these companies have become household names as their trailblazing mRNA vaccines became the first to be approved by regulators against the COVID-19 scourge. Their technology is based on synthetic messenger RNA – short for ribose nucleic acid – which is a short transcript of a longer DNA code.

Vaccination 110

Vaccination Vaccine Doctors Doctor 110

Drug Discovery World

FEBRUARY 6, 2024

However, many have cautioned against too great a reliance on these tools and emphasised the importance of checks and balances to ensure the systems are properly regulated and results are verified. The use of AI is rapidly developing in society and as regulators we see more and more applications in the field of medicines.

Marketing 69

Marketing Medicine Genome Genomics 69

Drug Discovery World

OCTOBER 18, 2023

What’s more, they are expensive, not readily available, and in many countries, there are a host of stringent regulations, as well as ethical and legal considerations around their use. Mouse models, on the other hand, are cost-effective and readily available, making them a favourite for many researchers. Accessed 26 Jul 23.

Gene Therapy 64

Gene Therapy Gene Clinical Trials Clinical Trials 64

Roots Analysis

AUGUST 31, 2023

Further, the expression of any gene is dependent on the rate at which it is transcribed into mRNA and translated into proteins. There are various regulatory proteins or transcription factors that are responsible for affecting the transcription rate. These genetic switches assist transcription factors in binding to the promoter region.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

The Pharma Data

OCTOBER 18, 2020

(NASDAQ:CDXC) today highlighted a new study published in The European Molecular Biology Organization Journal looking at the effect of nicotinamide riboside (NR) on maintaining telomeres, the protective regions at the end of DNA strands. Telomeres are “caps” at the end of chromosomes that protect DNA from getting worn away as cells replicate.

DNA 52

DNA Scientist Genetic Disease Research 52

Drug Discovery World

APRIL 14, 2023

He is being recognised for his revolutionary contributions to developing the first gene-edited cell-based therapy for cancer that involves the genetic re-engineering of a patient’s own T cells to combat their disease, and for demonstrating that adoptive T-cell therapy can induce remission and in some cases cure patients with advanced cancer.

Genetics 52

Genetics Research Medicine Clinical Research 52

XTalks

NOVEMBER 7, 2022

Liquid biopsy tests in oncology involve isolating entities such as circulating tumor cells (CTC), circulating tumor DNA (ctDNA) and tumor-derived exosomes. The cfDNA that originates from tumors is called circulating tumor DNA (ctDNA). These tumor-derived entities are used to derive genomic and proteomic data.

Clinical Trials 98

Clinical Trials Clinical Trials Trials FDA Approval 98

Drug Discovery World

JULY 11, 2023

Now, with the growth of publicly available genomics, transcriptomics, and proteomics databases, the ability to quickly carry out large-scale DNA, RNA, and protein screenings, and the availability of massive sets of de-identified patient data, the amount of high-value, analysable data has reached enormous proportions.

Big Data 40

Big Data Development Life Science Drugs 40

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. This is essential to its use as a therapeutic agent and gives the technology a vast versatility making it suitable to treating a wide range of diseases – especially those that have high protein expression.

RNA 52

RNA Protein Vaccination Vaccine 52

Drug Discovery World

JULY 29, 2022

Scientists from seven research institutions including the University of Oxford and the University of Edinburgh have used pioneering bioinformatic modelling to investigate the molecular interactions of the p53 protein known to give protection against cancers.

Gene 52

Gene DNA Protein Genetics 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. Epigenetic Editing with CRISPR. pyogenes dCas9.

DNA 98

DNA Gene Gene Silencing Genome 98

Drug Discovery World

JULY 15, 2022

The remaining 8% of the genome was often referred to as the dark matter of the genome or sometimes even ‘Junk DNA’ 2. The sequencing technology relied on could only yield sequences of relatively short DNA fragments, so the assembly of sequences from highly repetitive regions was a huge challenge.

Genome 52

Genome Genomics DNA Containment 52

Drug Discovery World

AUGUST 10, 2022

To overcome this evasion tactic, the CAR protein was developed to recognise other markers on cancer cells. CAR is a fusion protein composed of an extracellular antibody fragment that recognises antigens and an intracellular domain that directs the T cell to the tumour. Identifying the right CAR T cell target.

Manufacturing 98

Manufacturing Gene Genome Genomics 98

Drug Discovery World

MARCH 6, 2024

The development of effective new disease-modifying treatments in neurodegenerative and neuropsychiatric disorders has been hindered by their inherent genetic complexity, environmental influences, and clinical variability. This is comparatively easier to accomplish in these diseases because they are relatively monogenic.

Genome 64

Genome Genomics Clinical Trials Clinical Trials 64

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. In one example, Choi et al.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 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. parvovirus B19, Flaviviridae, Ebola, and Coronaviridae), DNA viruses (e.g., CRISPR diagnostics have successfully detected various pathogens, including RNA viruses (eg.

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

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. These therapeutics are broadly classified into two categories, namely coding RNAs and non-coding RNAs.

RNA 40

RNA Protein Genetics Gene Expression 40

pharmaphorum

SEPTEMBER 14, 2020

Last week geneticist Dr Charles Steward shared with us his experiences of searching for a genetic cause for his children’s rare neurological diseases. While there may be just under 20,000 confirmed protein coding genes, it turns out that much of the genome outside of these genes is also important in regulating how the genome is controlled.

Genome 118

Genome Genomics Medicine Gene 118

Drug Discovery World

MAY 11, 2023

Kerstin Pohl , Senior Global Marketing Manager, Gene Therapy & Nucleic Acid at SCIEX, looks at the application of liquid chromatography-mass spectrometry for analysing host cell proteins. Host cell proteins (HCPs) – proteins derived from the host cells used for viral production—are one class of process-related impurities.

Production 52

Production Gene Protein Vaccination 52

Drug Discovery World

AUGUST 1, 2023

1 August was first chosen as World RNA Day in 2018 as a play on AUG (adenine, uracil and guanine), a triple sequence of RNA (called a codon) that initiates protein synthesis by the cell. Since then, it has been observed to publicise the importance of this molecule in the generation of proteins in the body.

RNA 52

RNA Drugs Gene Silencing Vaccination 52

The Pharma Data

AUGUST 3, 2021

SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement. Spinal Muscular Atrophy (SMA) ?

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

The Pharma Data

JANUARY 10, 2021

People have done all kinds of things in genetics about genes that increase your risk and contribute to you getting Alzheimer’s. So, what you’re desperate for are some markers, that regardless of what’s causing your Alzheimer’s, still cause that protein level to change.”. . 2020 Was Educational for Industry & Regulators.

Vaccination 52

Vaccination Vaccine Regulation Drugs 52