XTalks

JULY 14, 2022

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. The proteins are interconnected and changes in one protein level can impact that of another.

Gene 98

Gene Regulation Protein Gene Editing 98

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

Drug Discovery World

MARCH 2, 2023

Epigenetics is still largely unexplored and referred to as the ‘dark matter’ of the genome. But the new findings answer a fundamental and longstanding question – how epigenetic proteins regulate the processes of transcription and gene expression, through which our genes are read and translated into proteins.

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. ZFPs can be engineered to make zinc finger nucleases, or ZFNs, which are proteins that can be used to edit genomes by knocking select genes in or out to specifically modify DNA sequences.

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

Drug Discovery World

MAY 17, 2024

Cancer trials are notorious because they are dependent on the genetic profile of patients and hence highly-selective. The researchers delved into the genetic makeup of tumours, recognising that genetic mutations heavily influence how tumours respond to drugs. The next challenge is related to the enrollment rates.

Trials 52

Trials Clinical Trials Clinical Trials Protein 52

XTalks

MARCH 18, 2024

By genetically editing microorganisms, scientists can now manipulate the metabolic pathways to produce foods with desirable properties. The precision fermentation study highlights how genetically engineered microorganisms can inhibit the growth of foodborne pathogens.

Genetic Engineering 52

Genetic Engineering Genetics Production Protein 52

Drug Discovery World

NOVEMBER 27, 2023

In addition, it has stimulated increased collaboration among drug developers, technology providers, supply chain organisations, and regulators. Engineering of Chinese hamster ovary cell lipid metabolism results in an expanded ER and enhanced recombinant biotherapeutic protein production. Biotechnol Bioeng 2012;118:2326.

Production 59

Production Engineer Gene Editing Gene 59

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 The combined actions of ivacaftor, tezacaftor and elexacaftor help hydrate and clear mucus from the airways.

Protein 52

Protein Gene Medicine Gene Therapy 52

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

pharmaphorum

JANUARY 20, 2021

A genomic analysis of the SARS-CoV-2 coronavirus, which causes COVID-19, shows that the virus accumulates around two mutations a month. The new variant identified in the UK in December shows significant mutations in the Spike protein and receptor binding domain that increase its infectivity.

Vaccination 52

Vaccination Vaccine Protein RNA 52

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. bluebird bio’s Philip Gregory.

Gene Therapy 57

Gene Therapy Gene Trials Gene Expression 57

Drug Discovery World

MARCH 7, 2023

More research work focuses on dissecting the genetic architecture of other diseases such atrial fibrillation and arrhythmia,” Dr Saad says. More research work focuses on dissecting the genetic architecture of other diseases such atrial fibrillation and arrhythmia,” Dr Saad says.

Biobanking 52

Biobanking Drugs Genome Genomics 52

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. Automation is also being applied to genomics sample preparation, an area that requires particular robustness and scalability.

Marketing 69

Marketing Medicine Genome Genomics 69

Drug Discovery World

MAY 5, 2023

Anthony Schmitt , PhD, Senior Vice President, Science at Arima Genomics, discusses the applications of 3D genomics in cancer research. But what about the patients whose genomic tests do not identify a cancer driver matched to an existing targeted therapy? with regards to detecting cancer driver mutations.

Genome 52

Genome Genomics Gene Genetics 52

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. Here he gives us a deeper look at how genomic medicine is evolving and the barriers that are preventing it from reaching its full potential.

Genome 118

Genome Genomics Medicine Gene 118

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

The Pharma Data

JULY 8, 2021

Today, access to the world’s largest browsable resource linking rare protein-coding genetic variants to human health and disease was launched through a genetic exome sequence analysis collaboration between AbbVie (NYSE: ABBV), Biogen Inc. Nasdaq: BIIB) and Pfizer (NYSE: PFE).

Genetics 52

Genetics Biobanking Protein Containment 52

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

MARCH 1, 2023

Avtar Singh is a Senior Scientist in the Cell and Tissue Genomics Department at Genentech, where he develops new approaches for image-based screening and high-throughput biological discovery. DDW’s Megan Thomas spoke with Singh about SLAS2023, where he presented Image-based screening of pooled genetic libraries.

Genetics 52

Genetics Genome Genomics Genotype 52

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

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

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

XTalks

NOVEMBER 7, 2022

These tumor-derived entities are used to derive genomic and proteomic data. Dr. Bahassi explained that technological advances in genetic sequencing of cfDNA have enabled liquid biopsies and led to marked increase in the detection of therapeutically-targetable mutations. Exosome-Based Liquid Biopsy in Oncology.

Clinical Trials 98

Clinical Trials Clinical Trials Trials FDA Approval 98

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

SEPTEMBER 5, 2022

Genome interpretation: To decipher genetic variants in cancer . The success of targeted therapies in cancer comes on the heels of identifying specific molecular targets resulting from genetic variations. When selecting patients for therapy, we currently look at variants that directly affect protein expression or function.

Development 52

Development Drugs Protein Genome 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

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

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. Cas12 protein creates staggered double-strand breaks (DSBs) and promotes the HDR mechanism instead of utilizing both non-homologous ends joining (NHEJ) and HDR.

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 through epigenetic editing without altering the genetic sequence itself. Epigenetic Editing with CRISPR.

DNA 98

DNA Gene Gene Silencing Genome 98

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

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 30, 2023

These RNA based therapeutics play a crucial role in protein production and regulation of gene functions. Self-amplifying RNA (saRNA): Self amplifying RNA is an engineered form of RNA, combining the amplifying genome (native to certain viruses) to the therapeutic gene of interest.

RNA 40

RNA Vaccination Vaccine Development 40

Drug Discovery World

MARCH 13, 2023

Whereas mature manufacturing systems for biologic drugs start with a consistent source material and use established platform processes, equipment, and analytics, cell therapy development represents just the opposite.  Consider a mature protein therapeutic that’s existed for 30-plus years. Genetic Engineering & Biotechnology News.

Gene Therapy 52

Gene Therapy Gene Manufacturing Development 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

Drug Discovery World

APRIL 17, 2023

There are a lot of tools that we know have reduced cancer death rates substantially for specific cancers, and if those are used more broadly, with greater access, we feel that this ambitious goal might be achievable,” said Dr Meredith Shiels of NCI’s Division of Cancer Epidemiology and Genetics, who led the study.

Research 52

Research Antibody Bioavailability Genetics 52

The Pharma Data

OCTOBER 28, 2020

The interim analysis did not include data from other exploratory outcome measures such as seizure frequency, sleep diaries, EEG patterns, UBE3A protein levels in the CSF, ambulation by wearable device, and adaptive behaviors. This condition is typically not inherited but instead occurs spontaneously. About Ultragenyx.

Protein 40

Protein Production Development Drugs 40