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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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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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. Optimise your cell therapy process: a guide to cell thawing. Free Whitepaper.

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Genetics Medicine Development Gene Therapy 328

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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RNA Gene Therapy Gene Protein 61

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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Protein RNA Scientist Manufacturing 95

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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Development Genetic Disease Genetics Gene Therapy 130

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. . Therapies that don’t precisely target diseased cells can have unintended effects in other healthy cells.” . Initial success in the lab.

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Gene Expression Gene Packaging Packaging 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. 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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Research Gene Therapy RNA Gene 59

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.

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Gene Expression Gene Genetic Disease RNA 59

Delveinsight

AUGUST 26, 2021

Shape Therapeutics inks gene therapy deal with Roche worth up to USD 3 Billion. Seattle biotech firm Shape Therapeutics has signed a deal potentially exceeding USD 3 billion with pharma giant Roche to bolster the development of gene therapies for Alzheimer’s and Parkinson’s disease.

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Vaccination Vaccine Gene Therapy RNA 98

XTalks

NOVEMBER 30, 2023

Its mechanism of action involves blocking the protein shell (capsid) of the HIV-1 virus, disrupting multiple crucial stages of the viral lifecycle. Over 26 weeks of Sunlenca combined with other antiretroviral drugs, 81 percent of participants achieved HIV RNA suppression, reaching levels low enough to be considered undetectable.

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Drug Discovery World

OCTOBER 4, 2022

This year’s event will cover some of the most exciting advancements in areas spanning screening, automation, high content imaging, disease models, cell and gene therapies and how innovation is being driven through partnerships and collaborations. Cell and gene therapies. billion, compared to $19.9

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Pharmaceutical Technology

DECEMBER 9, 2022

The move is particularly timely given the significant growth expected in global sales of cell and gene therapies. Beyond our expertise in small molecules, we have built a solid reputation with biopharmaceutical companies for the characterisation and analysis of monoclonal antibodies, therapeutic proteins and biosimilars.

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Drug Discovery World

FEBRUARY 23, 2024

Data on VY-TAU01, Voyager’s lead anti-tau antibody candidate, and on Voyager’s tau silencing gene therapy programme will be presented at the upcoming 2024 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (AD/PD 2024), taking place March 5-9, 2024, in Lisbon, Portugal.

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Gene Therapy Gene Antibody Protein 52

pharmaphorum

MARCH 9, 2022

billion takeover of Bioverativ in 2018 – is extended in the body by fusing it with von Willebrand factor (vWF), a protein that binds to factor VIII in the blood and makes it more stable, protecting it from degradation. The half life of the new drug – one of the main draws for Sanofi’s $11.6

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Protein Sales Gene Therapy Regulation 94

pharmaphorum

AUGUST 5, 2021

billion upfront to claim ownership of Vividion ‘s drug discovery platform, which uses chemoproteomics to identify new binding sites on proteins and find small-molecule compounds that interact with them, with another $500 million on the table if pipeline candidates meet targets. ” Bayer is paying $1.5 billion ($5.3

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DNA Gene Therapy Protein Gene 52

Drug Discovery World

APRIL 30, 2024

Reece Armstrong explores the potential and future opportunities for mRNA-based therapies. Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. We can see this versatility in the various treatments that are currently being researched.

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Drug Discovery World

OCTOBER 16, 2023

A company within this ecosystem is STORM Therapeutics, a clinical stage biotechnology company creating novel small molecule therapies that inhibit RNA modifying enzymes (RME) for use in oncology and other diseases. billion in funding.

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Drugs Life Science Gene Therapy Genome 75

pharmaphorum

NOVEMBER 24, 2021

Here’s a snapshot of our world class speaker line-up: Andreas Kuhn, Senior VP – RNA Biochemistry & Manufacturing, BioNTech . Elaine Peters, Director, Analytical – Individualized Cell & Gene Therapies, Genentech. Danilo Casimiro, CSO, Sanofi Pasteur. Patrick Finn, Senior Director – Rare Diseases, Moderna.

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Vaccination Vaccine Gene Therapy RNA 52

The Pharma Data

JANUARY 10, 2021

FBX-101 is a first-in-human AAV gene therapy. Krabbe disease is a rare and fatal pediatric leukodystrophy caused by mutations in the galactosylceramidase (GALC) gene. INZ-701 is a soluble, recombinant protein containing the extracellular domain of native human ENPP1 fused to the Fc domain of the immunoglobulin IgG1.

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pharmaphorum

JULY 11, 2022

Sanofi R&D in its core haemophilia category is advancing on three fronts – engineered proteins, RNA interference drugs and gene therapies – and highlighted results in two of those at the International Society on Thrombosis and Haemostasis (ISTH) congress over the weekend.

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Pharmaceutical Technology

DECEMBER 11, 2022

The first subject was dosed in the trial, which is designed to evaluate the safety, tolerability and immunogenicity of a nucleoside-modified RNA-based combination vaccine approach. 5 Omicron sublineages spike proteins. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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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.

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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.

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Manufacturing Vaccination Vaccine Production 52

Roots Analysis

MARCH 16, 2023

Messenger ribonucleic acid (mRNA) is a single-stranded molecule that is complementary to a gene’s DNA. It is important in the process of protein synthesis because mRNA is responsible for transferring genetic information from DNA to ribosomes, which then decodes the genetic information into a protein.

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Drug Discovery World

SEPTEMBER 28, 2022

DeepMind, a British AI company owned by Google which focusses on predicting proteins and has released the predicted structures of 350,000 proteins, covering almost the entire human body. . These vary from tiny start-ups to well-established industry players. Some recent highlights from market-leaders include: .

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Pharma Companies Drugs In-Vivo Genetics 52

Drug Discovery World

NOVEMBER 13, 2023

Ahead of protein and antibody engineering conference PEGS Europe 2024 in Lisbon, DDW’s Megan Thomas looks at what to expect from each track of the annual biologics technology meeting. Ali Madani, PhD, Founder and CEO, Profluent Bio, on: ‘Protein engineering with large language models’.

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Protein Antibody Engineer Scientist 59

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.

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Manufacturing Gene Genome Genomics 98

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

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DNA Genome Genomics RNA 98

Drug Discovery World

MAY 30, 2023

From antibody-drug conjugates and RNA, to CRISPR and CAR-T therapy, DDW asked the drug discovery industry what the most important development in oncology over the last five years has been. At PhoreMost, we have been particularly excited about the emergence of targeted protein degradation (TPD) as a new therapeutic modality.

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Research Drugs Protein RNA 52

Delveinsight

JANUARY 18, 2021

However, DelveInsight’s Angelman syndrome market analysis report estimates that several approaches focused primarily on the Gene Therapies, Topoisomerase Inhibitors, Cannabidiol, Protein Phosphatase 2A inhibitor, among others are under development to find a cure for the Angelman syndrome.

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Marketing Gene Drugs Protein 52

Drug Discovery World

OCTOBER 13, 2022

Prior to the pandemic, messenger RNA (mRNA) drug development was niche and predominantly pointed at different disease targets, including infectious diseases and cancers. One aspect that makes mRNA technology popular with drug developers is that its proteins can be easily edited. Why mRNA has potential .

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Drugs Manufacturing Vaccination Vaccine 52

Pharmaceutical Technology

FEBRUARY 1, 2023

A major breakthrough from the National Institutes of Health (NIH), in mapping the structure of the RSV protein significantly changed the landscape, allowing companies to leap into RSV drug research with better efficacy results. However, subsequent RSV drugs, like Medimmune’s motavizumab, failed in clinical trials.

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Vaccination Vaccine Antibody FDA Approval 162

Drug Discovery World

OCTOBER 17, 2023

Today, spatial omics technologies enable imaging-based identification of cells in their native tissue context while simultaneously detecting hundreds of RNAs or tens of protein markers in the same cells. The first step to reach this goal will be to generate protein and RNA data from the same sample.

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Gene Expression Drugs Protein Gene 52

The Pharma Data

DECEMBER 27, 2020

Galidesivir is a broad-spectrum antiviral, an adenosine nucleoside analog that blocks viral RNA polymerase. The drug is a topical ointment applied to the lower lid to address the build-up and shedding of proteins at the opening of the Meibomian gland. The drug is an autologous IL13Ralpha2-CAR-T cell product.

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Trials Antibody Gene Therapy Clinical Trials 52

Drug Discovery World

DECEMBER 15, 2022

Potential solutions: For CRISPR systems, the likelihood of off-target editing has been shown to be influenced by a variety of factors, ranging from the design of the gRNA 1 and Cas9 protein 2 , to the targeted cell type 3 , to name a few examples. DNA targeting specificity of RNA-guided Cas9 nucleases . In one example, Choi et al.

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Gene Editing Gene In-Vivo Genome 52

Drug Discovery World

JANUARY 23, 2023

In the engineering of more complex therapeutic solutions, multiple gene editing events are often required, raising the risk of additional unexpected cytotoxic/oncogenic effects through both indel formation, as well as chromosomal translocation events arising from multiple DSBs. Enter, base editing. Dis Model Mech. 2020;13:dmm042317.

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