The Pharma Data

MARCH 7, 2022

By combining CRISPR technology with a protein designed with artificial intelligence (AI), it is possible to awaken individual dormant genes by disabling the chemical “off switches” that silence them. The chemical modifications that regulate gene activity are called epigenetic markers. it can be reawakened.

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Protein Gene Genome Genomics 52

Medical Xpress

JANUARY 20, 2023

A team of researchers at the University of Texas Southwestern Medical Center has found that it is possible in mice to protect the heart from ischemia-reperfusion using the CRISPR-Cas9 gene editing system.

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Gene Editing Genome Genomics Gene 81

Drug Discovery World

SEPTEMBER 25, 2023

CRISPR gene editing companies Integrated DNA Technologies (IDT) and Aldevron have inked a new global distribution agreement to expand CRISPR products for cell and gene therapy customers. The post Partners working to realise the future of genomic medicine appeared first on Drug Discovery World (DDW).

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XTalks

JULY 14, 2022

Every gene in a cell is expressed at exact levels due to complex gene regulatory networks. For example, when T cells (a type of white blood cells that fight off infections and cancer) are activated in our immune systems, thousands of proteins in these cells change. Using CRISPR Technology to Disrupt Networks of Immune Genes.

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XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

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pharmaphorum

MARCH 19, 2021

Gain Therapeutics has priced its initial public offering, raising more than $40 million for its computational drug discovery platform and pipeline of drugs for diseases associated with protein misfolding. . The post Protein misfolding specialist Gain nets $40m from IPO appeared first on. and valuing the company at around $130 million.

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Protein Gene Editing Drugs Clinical Trials 100

Medical Xpress

DECEMBER 12, 2022

Ana Guadaño at the Alberto Sols Biomedical Research Institute (IIBM, a combined CSIC-UAM center) and involving the Complutense University of Madrid (UCM), used CRISPR gene editing techniques to incorporate into mice a mutation of the MCT8 protein responsible for transporting thyroid hormones to the interior of the cell.

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Gene Editing Genetics Hormones Medicine 75

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., “Accessing state-of-the-art LNP technology through this collaboration will add momentum to our gene editing efforts for the benefit of patients.”

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STAT News

SEPTEMBER 16, 2022

Preliminary results from the study — just the second to show that CRISPR-based gene editing can be delivered systemically and performed in vivo, or inside the body — found that the treatment, NTLA-2002, reduced levels of the disease-causing protein, kallikrein, by 65% and 92% in the low- and high-dose cohort, respectively.

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DNA Gene Editing In-Vivo Genetics 142

XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. It also affects Hispanic Americans, but at a lower prevalence.

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Drug Discovery World

NOVEMBER 14, 2023

A Phase Ib trial has shown low-density lipoprotein cholesterol (LDL-C) reductions up to 55% and blood PCSK9 protein reductions up to 84% after a single infusion of Verve Therapeutics’ Verve-101. It is caused by a single gene mutation that impairs the body’s ability to mediate LDL cholesterol.

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In-Vivo Gene Editing Gene Genome 52

Pharmaceutical Technology

AUGUST 26, 2022

ElevateBio has signed a long-term strategic collaboration with the University of Pittsburgh, US, to establish a biomanufacturing centre for expediting cell and gene therapy development. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Gene Editing Gene Manufacturing 162

pharmaphorum

APRIL 23, 2021

Vertex Pharma has ramped up its involvement in gene-editing medicines for the second time in a week, paying Obsidian Therapeutics $75 million upfront to access its technology platform. . For example, adding a small-molecule might stabilise the medicine, allowing levels of the target protein to increase.

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Gene Editing Genetics Drugs Gene 52

Drug Discovery World

DECEMBER 1, 2023

The key drug discovery news this week focuses on experimental approaches to the treatment of some of the most challenging diseases, including a mathematical model that can simulate Alzheimer’s progression and using CRISPR gene editing to ‘shred’ tumours. Can a plant-based discovery pave the way for new drug treatments?

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Drug Discovery World

OCTOBER 6, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

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Drugs Gene Editing Genome Genomics 52

STAT News

SEPTEMBER 16, 2022

Preliminary results from the study — just the second to show that CRISPR-based gene editing can be delivered systemically and performed inside the body — found the treatment, NTLA-2002, reduced levels of the disease-causing protein, kallikrein, by 65% and 92% in the low- and high-dose cohort, respectively.

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Gene Editing Genetic Disease Genetics Protein 94

Drug Discovery World

MARCH 22, 2023

The findings showed that targeting two regulators that control gene functions related to inflammation led to at least 10 times greater T cell expansion in models, resulting in increased antitumour immune activity and durability. In some mice, it also led to overproduction of lymphocytes, causing toxicity.

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Gene Editing Gene Engineer Regulation 52

Drug Discovery World

NOVEMBER 27, 2023

Upstream cell line development has seen notable progress with recent advances in cell line engineering, particularly in enhancing gene of interest integration specificity, clone selection, and production efficiency. Next-generation engineering tools, such as gene editing have enabled knock-out cell lines with enhanced characteristics.

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pharmaphorum

JANUARY 10, 2022

Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. CRISPR drugs can be used to modify the expression of disease-associated proteins in the body, for example, by correcting a mutation in a specific gene.

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Drug Discovery World

OCTOBER 31, 2022

The last month has seen huge strides forward in our understanding of cancers, particularly in how they develop resistance to therapies and how we can ‘outsmart’ them using gene editing or different therapeutic pathways, but also how we can better target drugs to individuals and accurately predict treatment outcomes.

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Pharmaceutical Technology

MARCH 1, 2023

The drug candidate targets DNA-dependent protein kinase (DNA-PK). Merck also provides a wide range of products including lab water systems, gene editing tools, cell lines, antibodies and end-to-end systems. The drug candidate is administered through the oral route in the form of capsule and tablet. It is a new chemical entity.

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Pharmaceutical Technology

MARCH 1, 2023

The drug candidate targets DNA-dependent protein kinase (DNA-PK). Merck also provides a wide range of products including lab water systems, gene editing tools, cell lines, antibodies and end-to-end systems. The drug candidate is administered through the oral route in the form of capsule and tablet. It is a new chemical entity.

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Gene Editing DNA Cosmetics Life Science 100

Drug Discovery World

FEBRUARY 16, 2023

Aera’s technology also includes a licensed therapeutic enzyme platform based on the discovery of novel, compact, and programmable gene editing enzymes. “Challenges remain with current delivery approaches, and genetic medicines applications have been generally restricted to the liver and ex vivo settings.

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Genetics Medicine Gene Editing In-Vivo 52

Pharmaceutical Technology

FEBRUARY 15, 2023

Cas, also called CRISPR associated proteins, are a family of nucleases such as Cas9, Cas12a, Cas12b, and CasX that process these sequences. The eukaryotic genome can be cut at any desired position by introducing plasmids containing Cas genes and specifically constructed CRISPRs into eukaryotic cells.

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WCG Clinical

NOVEMBER 17, 2023

Although the NIH Guidelines were originally written with non-clinical laboratory research in mind, they also apply to human gene transfer (HGT) research, wherein rsNA or rsNA-containing products are administered to research participants. Since then, however, certain genetic engineering technologies (e.g.,

Genome 52

Genome Genomics Gene Editing Genetic Engineering 52

pharmaphorum

SEPTEMBER 18, 2020

Vertex is already the top player in CF with a quartet of exon-skipping medicines that tackle the mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that underlies the disease, but there are still a sizeable group of patients with no treatment options.

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Drug Discovery World

JANUARY 12, 2023

The genome is the blueprint for life, consisting of genes made up of thousands of pairs of DNA bases. Altering one base pair can drastically change how the gene’s protein product functions, while mutations can lead to the development of a range of genetic disorders. . Limitations of organoids.

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Gene Editing Gene Drugs In-Vivo 98

Drug Discovery World

OCTOBER 24, 2023

A BioIndustry Association (BIA) report lists the widespread adoption of artificial intelligence (AI) and machine learning (ML), the advancement of gene editing technologies, the power of engineering biology, and a growing interest from major pharmaceutical players as key trends driving the sector.

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Drug Discovery World

DECEMBER 12, 2022

According to the publication, “A small clinical trial has shown that researchers can use CRISPR gene editing to alter immune cells so that they will recognise mutated proteins specific to a person’s tumours. Those cells can then be safely set loose in the body to find and destroy their target. . “It

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Drugs Gene Editing Genome Genomics 52

XTalks

JANUARY 26, 2024

The company is developing a variety of ingredients, from proteins to natural preservatives. It employs advanced techniques like molecular farming and gene editing. But Elo Life Systems is not just about sweeteners.

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Cloudbyz

MARCH 6, 2024

Understanding Biologics: Biologics are a class of therapeutic agents derived from living organisms, such as cells, tissues, or proteins. The Promise of Gene and Cell Therapies: Beyond monoclonal antibodies and protein-based biologics, the field of biotechnology is rapidly advancing towards the development of gene and cell therapies.

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Drug Discovery World

DECEMBER 13, 2022

One form of FCMD is caused by a genetic abnormality in the ‘fukutin’ (FKTN) gene. The genetic anomaly blocks the chemical glycosylation of a biologically important protein. The disease manifests itself in the form of a severe neuromuscular disorder. .

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RNA Genetics Gene In-Vitro 52

XTalks

OCTOBER 12, 2021

14-3-3 zeta) protein, which the UToledo researchers had been studying for its role in immune-related pathologies, such as aortic aneurysms and in signaling pathways involving the pro-inflammatory cytokine interleukin-17. However, they found the opposite to be true, where the protein was actually protective against development of the disease.

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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. These findings signify a significant step forward in the development of AGT103-T as a potential gene therapy for HIV.

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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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Gene Editing Gene In-Vivo Genome 52

Roots Analysis

JANUARY 16, 2023

You may also be interested in the following titles: Targeted protein degradation market , 2022-2035. Gene Editing beyond CRISPR Market, 2022-2035. Cell and Gene Therapy Bioassay Services Market, 2021-2030. Our Social Media Platform. Facebook – [link]. LinkedIn – [link]. Twitter – [link].

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Bioassay Gene Editing Marketing Gene Therapy 52

pharmaphorum

JULY 13, 2021

In ATTR amyloidosis, transthyretin (TTR) protein misfolds and accumulates as amyloid deposits throughout the body. In 2019, for example it agreed to work with Dicerna on gene-silencing for liver-related cardio-metabolic disease in a deal that involved a $225 million upfront payment.

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