Scienmag

NOVEMBER 11, 2020

Credit: Masato Kanemaki Researchers can now more accurately and precisely target specific proteins in yeast, mammalian cells and mice to study how knocking down specific protein traits can influence physical manifestation in a cell or organism. “Conditional gene knockout and small interfering RNA […].

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Drug Discovery World

AUGUST 16, 2023

Samir Ounzain , PhD, CEO & Co-Founder of HAYA Therapeutics, looks at how a better understanding of our DNA can lead to increased activity for RNA therapeutics. The whole world realised the power of RNA when the Covid-19 pandemic brought us the first mRNA-based vaccines.

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BioSpace

SEPTEMBER 22, 2020

RNA-binding proteins are emerging as the next generation of molecular medicine.

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Drug Discovery World

NOVEMBER 1, 2022

Researchers say they have successfully used a cell’s natural process for making proteins to ‘slide’ genetic instructions into a cell and produce critical proteins missing from those cells. . Two methods currently used to deliver protein-making packages into cells, ‘mini promoters’ and serotype-mediated gene expression, vary widely.

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pharmaphorum

JULY 20, 2022

Armed with a $100 million second-round financing, CAMP4 Therapeutics is preparing to start the first clinical trial of a drug targeting regulatory RNA (regRNA) molecules that can be used to fine-tune the expression of genes. ” The post CAMP4 raises $100m to take lead RNA drugs into clinic appeared first on. .

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Scienmag

AUGUST 4, 2020

Led by Boyce Thompson Institute’s Andrew Nelson, four partners will identify RNA modifications and develop resources that may lead to hardier crops Credit: Photo credit: Anna Nelson Dittrich ITHACA, NY, August 4, 2020 — RNA perform a variety of functions in cells, helping with everything from regulating genes to building proteins.

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Pharmaceutical Technology

JANUARY 19, 2023

AOC 1020 has been designed for the treatment of the underlying cause of FSHD, which is caused by the abnormal expression of a gene known as double homeobox 4 or DUX4. This DUX4 protein abnormal expression leads to modifications in gene expression in muscle cells which are associated with progressive muscle function loss in FSHD patients.

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Drug Discovery World

JANUARY 10, 2023

CircRNAs represent an emerging, powerful mechanism for delivering therapeutics and vaccines due to their protein-coding potential and improved stability in comparison to their linear mRNA counterparts. . The post Alliance to develop circular RNA-based therapeutics appeared first on Drug Discovery World (DDW).

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Pharmaceutical Technology

APRIL 20, 2023

Targovax has unveiled plans to rebrand as Circio , reflecting its strategic shift to focus on expediting the development of its innovative circular RNA (circRNA) platform. Initially reported in 2011, CircRNA is a naturally occurring class of RNA. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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BioSpace

SEPTEMBER 15, 2020

The company is genetically programming RNA not just to deliver a gene of interest, but to control the location, timing and intensity of therapeutic protein expression using mRNA-encoded logic circuits.

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Scienmag

FEBRUARY 10, 2022

Also known as transcriptional activators for their ability to induce transcription of genes into RNA messages, these proteins are essential for the cells to function properly. Yet little is known about these proteins, and it wasn’t clear how many activators there might be in human cells – until now.

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Scienmag

APRIL 27, 2022

CHAPEL HILL, NC – Inside embryonic cells, specific proteins control the rate at which genetic information is transcribed from DNA to messenger RNA – a crucial regulatory step before proteins are created. Those specific “regulatory” proteins are called transcription factors, and they do their thing by binding to […].

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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. and Nelson D.

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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. DS: You describe your therapeutics as RNA Amplifiers. How do they work?

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Medical Xpress

FEBRUARY 9, 2023

When cells copy DNA to produce RNA transcripts, they include only some chunks of genetic material known as exons and throw out the rest. The resulting product is a fully-mature RNA molecule, which can be used as a template to build a protein.

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Scienmag

MARCH 17, 2022

Cells use RNA as a versatile tool to regulate the activity of their genes. Small snippets of RNA can fine-tune how much protein is produced from various genes; some small RNAs can shut genes off altogether.

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pharmaphorum

JANUARY 6, 2022

Roivant is the latest pharma group to take a position in the emerging field of therapies targeted at RNA splicing, licensing exclusive rights to a drug developed by a unit of Japanese drugmaker Eisai. . The post Roivant joins RNA splicing push with Eisai deal appeared first on.

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BioTech 365

FEBRUARY 25, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Super-resolution RNA imaging in live cells.Ribonucleic acid (RNA) is key to various fundamental biological processes. It transfers genetic information, translates it into proteins or supports gene regulation.

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XTalks

JUNE 20, 2022

Alnylam Pharmaceuticals, a leading RNA interference (RNAi) therapeutics biopharmaceutical company, announced it received approval from the US Food and Drug Administration (FDA) for its RNAi therapeutic Amvuttra (vutrisiran) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (ATTR) amyloidosis in adults.

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Pharmaceutical Technology

DECEMBER 23, 2022

This alliance is utilising the Axiomer ribonucleic acid (RNA) editing platform of ProQR to address ailments affecting the liver and nervous system. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. The companies entered the initial agreement in September last year. Free Whitepaper.

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Scienmag

MAY 7, 2021

Credit: UPMC PITTSBURGH, May 7, 2021 – In a paper published today in Nature Communications, an international group of collaborators led by researchers at UPMC Children’s Hospital of Pittsburgh have identified a genetic cause of a rare neurological disorder marked by developmental delay and loss of coordination, or ataxia.

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The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., This delivery technology protects the messenger RNA (mRNA) payload after administration allowing it to be safely and effectively delivered into cells.

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Scienmag

MAY 5, 2021

Transcription is a vital process in bacterial cell, where genetic information in DNA is transcribed to RNA for the translation of proteins that perform cellular function. Credit: The Hong Kong University of Science and Technology The emergence and spread of new forms of resistance remains a concern that urgently demand new antibiotics.

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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.,

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Scienmag

MARCH 4, 2021

Findings explain how plants use RNA to defend against fungal invaders Credit: Nicole Ward Gauthier/University of Kentucky New research reveals an essential step in scientists’ quest to create targeted, more eco-friendly fungicides that protect food crops.

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Drug Discovery World

MARCH 26, 2024

For example, progress made in developing gene mutant-specific inhibitors, antibody-drug conjugates (ADC), or cellular therapies. We were excited to unveil data showing that two distinct chemical series exhibit METTL1 inhibition in vitro at low nanomolar concentrations with minimal interference with other RNA and protein methyltransferases.

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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. When it gives the green light, H3K4me3 allows RNA polymerase II to move along DNA, transcribing it into RNA as it moves.

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Scienmag

JUNE 29, 2022

Myotonic dystrophy type 1 (DM1) is the most common form of muscular dystrophy, characterized by progressive muscle wasting and weakness and caused by abnormally repetitive DNA segments that are transcribed into toxic molecules of RNA. Rett […].

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pharmaphorum

JANUARY 6, 2021

Two years after starting to work together, Novo Nordisk and Dicerna have selected the first candidate from a joint project to find new, gene-silencing drugs for liver-related cardiometabolic diseases. The post Novo Nordisk’s gene silencing alliance with Dicerna bears first fruit appeared first on.

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XTalks

OCTOBER 21, 2020

The answer may lie in the genetic code of the virus, which scientists at Duke University have found contains several silent mutations that affect protein folding. The researchers focused on the spike proteins that protrude from the surface of the coronavirus, which are responsible for viral attachment and entry into host cells.

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Scienmag

JULY 1, 2021

Almost twenty years ago, the process of RNA silencing was discovered in plants, whereby small fragments of RNA inactivate a portion of a gene during protein synthesis.

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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. Successful industry/academia collaboration Fewer than 5% of rare diseases have approved therapies and most of these treatments use gene therapy.

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Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. million price point per dose.

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BioSpace

OCTOBER 19, 2020

Through their work, they were able to determine that the protein, TAp63, appears to impact the level of RNA molecules within the body. This subsequently connects the activities of p53 and AKT, the altered genes in cancer.

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Scienmag

MAY 27, 2021

Capturing how RNA polymerase enzymes kick off transcription On scales too small for our eyes to see, the business of life happens through the making of proteins, which impart to our cells both structure and function.

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Pharmaceutical Technology

APRIL 26, 2023

BioMarin senior vice-president and chief scientific officer Kevin Eggan stated: “Activating RNA therapeutics have the potential to change the way we treat certain genetic diseases, particularly those characterised by the limited production of key proteins. Financial details of the deal were not divulged.

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Drug Discovery World

JANUARY 20, 2023

Precision medicine uses information about an individual’s genes, proteins, and environment to prevent, diagnose, and treat diseases, including cancer. Differences in DNA, RNA, and protein can be identified in individual tissue samples, and even in individual cells.

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