Drug Discovery World

APRIL 17, 2024

DS: CAMP4 leverages research from co-founder Richard Young on how regulatory RNA (regRNA) molecules control the expression of genes. JMB: Regulatory RNAs (or regRNAS) are molecules that directly control the expression of nearby protein-coding genes. DS: You describe your therapeutics as RNA Amplifiers. How do they work?

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Gene Expression Gene Genetic Disease RNA 59

The Pharma Data

MARCH 23, 2022

Polymerase is a viral protein that directs how Ebola virus replicates its genome as it infects new hosts. The researchers discovered that Ebola virus polymerase hijacks a cellular protein called GSPT1. To treat Ebola virus infections, researchers are taking a close look at a key piece of the virus: polymerase.

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Protein Genome Genomics Research 52

Drug Discovery World

MARCH 2, 2023

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. The team found that H3K4me3 acts like a traffic light at a busy intersection.

Gene Expression 52

Gene Expression Gene DNA RNA 52

Drug Discovery World

JANUARY 11, 2024

Researchers have used messenger RNA (mRNA) to create an effective therapy for a rare liver disease in preclinical studies, demonstrating the technology’s potential therapeutic use in people. Patients affected by the disease are found to also experience an imbalance of glutathione regulation, which is important for liver detoxification.

Research 59

Research Gene Therapy RNA Gene 59

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.

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DNA Genetics Gene Medicine 115

Delveinsight

SEPTEMBER 23, 2021

Boehringer stated that the Abexxa’s generation aims at cancer-specific proteins situated throughout the mobile versus proteins at the mobile membrane broadening the selection of possible cancer antigen objectives. 858 Therapeutics closes USD 60 Million series A round to drug RNA modulation. Jeffrey Stafford, Ph.D.,

RNA 61

RNA Gene Therapy Gene Protein 61

pharmaphorum

DECEMBER 1, 2020

Pfizer and BioNTech have filed their COVID-19 vaccine with the European medicines regulator, paving the way for a potential approval before the end of the year. The EMA has also confirmed it has received a filing from Moderna, which is producing a rival vaccine based on similar RNA technology.

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pharmaphorum

NOVEMBER 18, 2021

The two companies have been working together since 2019 to develop multiple RNA interference (RNAi) candidates for disorders such as non-alcoholic steatohepatitis (NASH), type 2 diabetes, obesity and rare diseases. billion in cash.

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Gene Silencing RNA Protein Gene 52

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).

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FDA Approval Drugs In-Vivo In-Vitro 52

pharmaphorum

OCTOBER 6, 2020

The announcement comes just days after the regulator said its CHMP scientific committee had begun to look at the first batch of data from AZ’s rival. The vaccine is based on different technology from AZ’s rival: while AZ’s uses a weakened and genetically modified cold virus to prime the immune system, BNT162b2 is based on a string of RNA.

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Vaccination Vaccine RNA Immune Response 58

pharmaphorum

JANUARY 20, 2021

The new variant identified in the UK in December shows significant mutations in the Spike protein and receptor binding domain that increase its infectivity. The variant that originated in South Africa carries a mutation in the Spike protein that has been shown to reduce antibody recognition and could therefore affect efficacy of new vaccines.

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Vaccination Vaccine Protein RNA 52

Roots Analysis

OCTOBER 30, 2023

In the past few years, Next Generation RNA therapeutics have emerged as one of the key therapeutic modalities in the modern healthcare industry. These RNA based therapeutics play a crucial role in protein production and regulation of gene functions.

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RNA Vaccination Vaccine Development 40

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.

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Vaccination Vaccine Doctors Doctor 110

XTalks

APRIL 6, 2022

XTALKS WEBINAR: Using CRISPR interference (CRISPRi) Viability Screens to Map Long Noncoding RNA Dependencies in Tumor Cells. Register for this webinar to get an overview of the role of long non-protein coding RNAs (lncRNAs) in gene regulations. Live and On-Demand: Thursday, April 21, 2022, at 11am EDT (4pm BST/UK).

DNA 105

DNA In-Vitro Doctors Doctor 105

pharmaphorum

NOVEMBER 9, 2020

Results will be discussed with regulators worldwide and the European Medicines Agency has already begun its rolling review of data to hasten a decision on the clinical trial dossier. The independent data monitoring committee that conducted the interim analysis said that there had been no serious safety concerns identified.

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Vaccination Vaccine Clinical Trials Clinical Trials 98

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. Its job is to carry coding information that is essential to the translation and processing of functional proteins.

RNA 52

RNA Protein Vaccination Vaccine 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. Non-coding RNAs include antisense oligonucleotides (ASOs) and RNA aptamers.

RNA 40

RNA Protein Genetics Gene Expression 40

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

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

Drug Discovery World

SEPTEMBER 28, 2022

Acts such as General Data Protection Regulation (GDPR) in the UK and the Health Insurance Portability and Accountability Act (HIPAA) in the US protect users’ health data from being used for commercial purposes. Another big problem looms for organisations attempting to access these private data sets; ever-stricter data privacy laws.

Pharma Companies 52

Pharma Companies Drugs In-Vivo Genetics 52

The Pharma Data

MARCH 5, 2021

This will be adapted to the needs of messenger RNA vaccine production for CureVac’s CVnCoV, the manufacturing of which is highly complex. At the Kundl site, Novartis is a pioneer and has decades of experience in pharmaceutical production of proteins and in more recent years of nucleic acids. Disclaimer.

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Manufacturing Vaccination Vaccine Production 52

STAT News

MARCH 6, 2023

Cambridge-based Moderna recently reported encouraging results from a study of another type of personalized cancer vaccine it developed with the pharmaceutical giant Merck; it uses messenger RNA technology to target advanced melanoma. Neoantigen vaccines are designed to target certain proteins called neoantigens on tumor cells.

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Vaccination Vaccine Drugs Clinical Trials 98

The Pharma Data

AUGUST 30, 2021

As the first and only small interfering RNA to provide effective and sustained LDL-C reduction, Leqvio helps manage a critical cardiovascular risk factor for ASCVD. Leqvio is the first and only approved small interfering RNA (siRNA) LDL-C-lowering treatment in Europe 6,7. It is currently under review by the U.S. a statin or ezetimibe).

RNA 52

RNA Trials Production Containment 52

Drug Discovery World

MARCH 1, 2023

DDW’s Megan Thomas spoke with Singh about SLAS2023, where he presented Image-based screening of pooled genetic libraries. This is a challenging way to conduct large genetic screens because it requires careful handling of thousands of samples, often using specialised automation, and introduces batch effects across samples.

Genetics 52

Genetics Genome Genomics Genotype 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.

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Big Data Development Life Science Drugs 40

XTalks

NOVEMBER 7, 2022

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. Exosomes are extracellular vesicles that contain molecular cargoes (such as various proteins, RNAs and DNAs) specific to the origin cell.

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Clinical Trials Clinical Trials Trials FDA Approval 98

The Pharma Data

DECEMBER 18, 2020

The Moderna COVID-19 Vaccine contains messenger RNA (mRNA), which is genetic material. The vaccine contains a small piece of the SARS-CoV-2 virus’s mRNA that instructs cells in the body to make the virus’s distinctive “spike” protein. View original content to download multimedia: [link]. SOURCE U.S.

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Vaccination Vaccine Production Manufacturing 52

Drug Discovery World

MAY 5, 2023

Efforts to fulfil the promise of precision medicine, including the use of targeted cancer therapies, necessitate that patients be treated based on the specific genetic alterations that drive their individual cancers. Since the genes are fused, the chimeric RNA transcripts encode different proteins, altering their function.

Genome 52

Genome Genomics Gene Genetics 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

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.

DNA 98

DNA Genome Genomics RNA 98

Delveinsight

JANUARY 18, 2021

Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system. 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.

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Marketing Gene Drugs Protein 52

The Pharma Data

JANUARY 21, 2021

Luckily, the new variants still rely on the coronavirus’ “spike protein” to infect cells, and the two COVID vaccines now on the U.S. market specifically target the spike protein to prevent transmission, explained Dr. Kathryn Edwards, scientific director of the Vanderbilt University Vaccine Research Program in Nashville.

Vaccination 52

Vaccination Vaccine Protein Immune Response 52

pharmaphorum

SEPTEMBER 21, 2020

A group of rare genetic disorders, FCS affects between 55 and 110 people in England and causes very high levels of triglyceride fat in the blood. The drug works by reducing the production of ApoC-III, a protein that regulates plasma triglycerides.

Drugs 79

Drugs RNA Medicine Genetics 79

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

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.

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DNA Gene Gene Silencing Genome 98

The Pharma Data

SEPTEMBER 8, 2020

About Moderna Moderna is advancing messenger RNA (mRNA) science to create a new class of transformative medicines for patients. For more information, visit www.merck.com and connect with us on Twitter , Facebook , Instagram , YouTube and LinkedIn. For more information, visit www.novavax.com and connect with us on Twitter and LinkedIn.

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Vaccination Vaccine Containment Clinical Trials 52

The Pharma Data

SEPTEMBER 8, 2020

is advancing messenger RNA (mRNA) science to create a new class of transformative medicines for patients. mRNA medicines are designed to direct the body’s cells to produce intracellular, membrane or secreted proteins that can have a therapeutic or preventive benefit and have the potential to address a broad spectrum of diseases.

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Vaccination Vaccine Containment Clinical Trials 52