The Pharma Data

JUNE 14, 2023

Scientists discover small RNA that regulates bacterial infection People with weakened immune systems are at constant risk of infection. Abell Chair in Molecular and Cellular Biology – and Pengbo Cao, a postdoctoral researcher in Whiteley’s lab, discovered a gene that drives the switch.

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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.

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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. JMB: Rick Young’s publication shows that RNA binding TFs fine-tunes gene expression.

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Scienmag

MARCH 19, 2021

Credit: Thomas Gorochowski In a recent study led by the University of Bristol, scientists have shown how to simultaneously harness multiple forms of regulation in living cells to strictly control gene expression and open new avenues for improved biotechnologies.

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Drug Discovery World

FEBRUARY 27, 2024

BS: Genetic tuning, also called epigenetic editing, actively manipulates the epigenetic machinery to influence gene transcription. Orchestrating the naturally occurring conductors of gene expression can shift the functional state of cells and alter gene expression.

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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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Drug Discovery World

FEBRUARY 14, 2024

A class of molecules playing a crucial role in the regulation of gene expression and other cellular processes can restore normal skin structure rather than producing a scar, a University of Manchester-led study suggests. We expected the removal of microRNA-29 would help outer layers of the skin to grow faster.

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Gene Expression Regulation Protein Gene 52

Drug Discovery World

FEBRUARY 16, 2024

Small RNAs promote wound-healing without scarring A class of molecules playing a crucial role in the regulation of gene expression and other cellular processes can restore normal skin structure rather than producing a scar, a University of Manchester-led study suggests.

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Scienmag

JUNE 14, 2021

The study, “Transcriptomics identifies STAT3 as a key regulator of hippocampal gene expression and anhedonia during withdrawal from chronic alcohol exposure,” is published in the journal Translational Psychiatry by researchers […].

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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

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. Through transcriptional profiling, they found that 141 genes significantly correlated with immobility, with 111 upregulated and 30 downregulated. “We

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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.

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pharmaphorum

OCTOBER 19, 2022

The dark genome – a loose term that covers non-coding regions of the genome that are capable of regulating the expression of genes, previously rather inaccurately referred to as junk DNA – is increasingly being explored by biopharma companies for new drug targets.

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pharmaphorum

JANUARY 13, 2022

AstraZeneca has expanded its efforts to develop new therapies targeting transcription factors – proteins that regulate processes in the cell that have become a hot topic for drug discovery in cancer and other diseases.

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Drug Discovery World

SEPTEMBER 27, 2023

While genetics has made significant strides in healthcare, the field of epigenetics unveils a new dimension in precision medicine, offering insights into the dynamic interplay between genes and experiences that shape our health and disease.

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Delveinsight

JULY 24, 2021

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. It is designed to target and inhibit the molecule that silences the paternal copy of the gene, called UBE3A antisense transcript (UBE3A-AS).

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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.

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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

pharmaphorum

NOVEMBER 22, 2022

TCFs – which control gene expression and can regulate important cellular processes, including cell growth and survival – have traditionally been held to fall into the undruggable category, with fewer than 1% of them successfully targeted for therapeutics.

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Drug Discovery World

AUGUST 1, 2023

Towards the end of last year, Kite, a Gilead company, entered into an exclusive license agreement with synthetic biology company Refuge to access its gene expression platform to develop potential treatments for blood cancers.

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XTalks

MAY 4, 2021

These modifications regulate gene expression without changing the sequence or structure of DNA. Now we have a simple tool that can silence the vast majority of genes,” said Jonathan Weissman, PhD, Whitehead Institute member, co-senior author of the new paper and a former UCSF faculty member in a statement from UCSF. “We

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The Pharma Data

MARCH 3, 2022

In 2018, Shalev and colleagues reported the benefits of verapamil in a one-year clinical study of Type 1 diabetes patients, finding that regular oral administration of verapamil enabled patients to produce higher levels of their own insulin, thus limiting their need for injected insulin to regulate blood sugar levels.

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pharmaphorum

JANUARY 29, 2021

California-based Sangamo Therapeutics is one such company that believes in the powerful potential of in vivo genome editing and regulation, together known as genome engineering, and has built up a sizable preclinical pipeline of genome regulation treatments for diseases such as Huntington’s disease and Amyotrophic lateral sclerosis (ALS).

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XTalks

MARCH 13, 2024

Secondly, other funded research is exploring differential gene expression regulated by DNA methylation. By identifying genes affected by DNA methylation in glaucoma, researchers hope to uncover new insights into the disease’s molecular mechanisms.

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pharmaphorum

FEBRUARY 9, 2021

InteRNA is focusing on the development of gene-silencing drugs based on microRNA, naturally occurring, non-coding strands of RNA that are thought to regulate gene expression in cells.

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Trials Gene Silencing RNA Gene Expression 57

The Pharma Data

JULY 11, 2021

On the one hand, they can boost the metabolism of T cells by inducing central regulators of energy metabolism. On the other hand, they can inhibit specific enzymes that regulate the accessibility to the genetic material and thus the gene expression in the T cells. In doing so, they induce epigenetic changes.

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Bacteria Genetics Regulation Scientist 52

XTalks

MAY 4, 2021

These modifications regulate gene expression without altering the sequence or structure of DNA. Now we have a simple tool that can silence the vast majority of genes,” said Jonathan Weissman, PhD, Whitehead Institute member, co-senior author of the new paper and a former UCSF faculty member in a statement from UCSF. “We

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DNA Gene Gene Silencing Genome 52

The Pharma Data

MARCH 7, 2022

The new technique controls gene activity without altering the DNA sequence of the genome by targeting chemical modifications that help package genes in our chromosomes and regulate their activity. The chemical modifications that regulate gene activity are called epigenetic markers.

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Protein Gene Genome Genomics 52

Roots Analysis

AUGUST 31, 2023

Type of Gene Regulation Based on the type of transcription factor ( activators / repressors ), gene regulation has been categorized into positive and negative gene regulation. When activator binds to the operon, it either speeds up or permits gene expression.

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The Pharma Data

SEPTEMBER 2, 2021

” With a particular focus on the lung, the researchers performed global gene expression analyses of the metastatic niche.

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Gene Expression Scientist Antibody Research 52

Roots Analysis

JANUARY 14, 2024

Like all drugs, biologics are regulated by the FDA. Usually, the desired gene, such as human insulin gene, when inserted into the plasmid of the host cell uses transcriptional and translational machinery of the host to express itself. They are different from small molecules in terms of their size and complexity.

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Delveinsight

JANUARY 18, 2021

For instance, GTX-102 , an antisense therapy silencing the gene expression by binding to the RNA molecule behind the regulation of its expression, has also secured USFDA Fast Track Designation. Although, it can be relieving that other players turned to different approaches for finding a cure for Angelman syndrome.

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Drug Discovery World

APRIL 30, 2024

Patients with argininosuccinic aciduria can also experience an imbalance of glutathione regulation, which is important for liver detoxification. Rare diseases are largely treated using gene therapies which employ modified viruses to bring the therapy to the disease cells.

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XTalks

DECEMBER 24, 2020

BioNTech/Pfizer’s vaccine became the world’s first approved COVID-19 vaccine, with UK regulators becoming the first to authorize it for emergency use, followed by Health Canada, which fully approved it. Among these, single-cell RNA sequencing (scRNA-seq) is particularly useful for analyzing the transcriptome with respect to gene expression.

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Drug Discovery World

JULY 18, 2023

Current research is focused on designing CRISPR-enhanced CAR T cells that are resistant to immunosuppressive cytokines such as transforming growth factor-β (TGF-β) 2 , programmed cell death protein (PD-1) 3 , or other negative T cell regulators 4 (CTLA-4, LAG-3, and TIM-3), thus improving anti-tumour functions.

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DNA Genome Genomics RNA 98

The Pharma Data

OCTOBER 15, 2020

Manual manipulation affects gene expression,” he pointed out, so minimizing that, as well as the vast quantities of plastics typically required, results in a more controlled process and a more consistent product. The advantage is that the cells exist in a very homogenous space and are 99% pure.”.

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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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