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

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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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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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 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

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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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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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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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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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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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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Drug Discovery World

MAY 15, 2024

In vivo proof-of-concept for Circio Holding’s differentiated circVec platform approach to gene therapy has been demonstrated in two posters at the American Society of Gene & Cell Therapy (ASGCT) 2024 annual meeting. The in vivo data showed statistically significant higher expression level and durability for circVec 2.1

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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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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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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. Cambridge is a focal point for many pharmaceutical companies working in this therapeutic area.

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Roots Analysis

AUGUST 31, 2023

Overview of Gene Switch The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E. Gene switches are sites on genes where regulatory molecules can bind to trigger transcription process, leading to expression of a particular gene.

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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

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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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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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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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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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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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

DECEMBER 15, 2022

As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Early phase clinical trials for gene editing therapies.

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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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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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Delveinsight

JANUARY 18, 2021

Angelman syndrome is a result of the missing or dysfunctional gene known as UBE3A. The gene is responsible for the normal and healthy development of an individual physically as well intellectually. GeneTx and Ultragenyx are co-developing the therapy.

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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. Another approach to address the limited availability of CAR T therapies consists of using CRISPR technology to generate CAR T-cell therapeutics derived from inducible pluripotent stem cells (iPSCs) 5.

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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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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. It’s a great tool for controlling gene expression.”. pyogenes dCas9.

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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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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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Drug Discovery World

JANUARY 23, 2023

Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies. Adult fibroblasts were cultured in a cocktail of transcription factors to reactivate embryonic gene expression patterns 2.

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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. Analysing spatial changes during disease progression or treatment response is key to fully understand pathologies.

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