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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BioTech 365

DECEMBER 1, 2020

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Genomics, gene-editing and the Blue Revolution.Aquaculture—the farming of fish and shellfish, is the world’s fastest growing primary industry.

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STAT News

NOVEMBER 4, 2022

A man with muscular dystrophy who was first in line to receive an experimental gene editing therapy tailor made to treat the cause of his rare form of the disease has died. The clinical trial was to be the first time anyone got a gene editing therapy for muscular dystrophy.

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Pharmaceutical Technology

AUGUST 26, 2022

This centre will be equipped with its supporting technologies, such as gene editing induced pluripotent stem cell (iPSC) and cell, vector and protein engineering expertise.

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pharmaphorum

JUNE 28, 2021

So far, human trial data for gene-editing drugs have been generated for ex vivo therapies, meaning the CRISPR technology is used to modify cells outside the body, which are then reinfused into the patient. — Eric Topol (@EricTopol) June 26, 2021. . — Eric Topol (@EricTopol) June 26, 2021.

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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

pharmaphorum

FEBRUARY 3, 2021

Dr Jennifer Harbottle, senior scientist in the R&D Base Editing team of PerkinElmer’s Horizon Discovery business, looks at progress made in the realms of biotechnology and next-generation diagnostics, vaccines and therapeutics, including the application of CRISPR-Cas9 gene editing in developing and refining cell therapies.

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Pharmaceutical Technology

FEBRUARY 13, 2023

This is particularly the case for pediatric forms of epilepsy where gene editing techniques like Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) are becoming more important, says Ferraro. Stoke Therapeutics is working on an antisense oligonucleotide called STK-001.

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Research Gene Gene Therapy Genome 264

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

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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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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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. These methyl groups must be refreshed so if PRC2 is blocked the genes it has silenced. it can be reawakened.

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Protein Gene Genome Genomics 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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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

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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Vaccination Vaccine Research Immune Response 105

Roots Analysis

FEBRUARY 15, 2022

Presently, there are several companies and universities, which are exploring the potential of different gene editing technologies beyond CRISPR for basic research, and the development of gene editing solutions. Genome Editing is a way of making changes in the DNA. Type of Genome Editing: Zinc-Finger Nuclease.

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XTalks

JULY 14, 2022

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. The proteins are interconnected and changes in one protein level can impact that of another.

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The Pharma Data

NOVEMBER 22, 2020

Scientists in Israel have used the CRISPR Cas-9 gene editing system to destroy cancerous cells in mice without damaging other cells. The product is the most advanced gene-editing approach in development for transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease. Photo courtesy of Science Advances.

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Gene Editing DNA Genome Genomics 52

XTalks

JANUARY 4, 2024

Hemophilia B, also known as congenital Factor IX (FIX) deficiency or Christmas disease, is a rare genetic bleeding disorder caused by a deficiency or defect in the FIX clotting protein. Treatment involves replacing the missing FIX clotting protein, traditionally through regular infusions.

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Gene Therapy Gene Gene Editing Sales 111

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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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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pharmaphorum

AUGUST 31, 2022

One area of the gene therapy space that could become a major treatment modality is CRISPR technology. The key to why this technology could become central to gene editing is due to the specificity with which it can edit DNA and that it is also a relatively cheap process, compared with existing technologies.

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Gene Therapy Gene Gene Editing DNA 98

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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WCG Clinical

NOVEMBER 17, 2023

This change expanded the definition of HGT research to include research using nucleic acids that are able to replicate, be transcribed, translated into protein, and/or integrate into the host genome. If implemented, these changes would broaden the definition of HGT to include new gene editing and genome-modified products.

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Genome Genomics Gene Editing Genetic Engineering 52

pharmaphorum

JANUARY 10, 2022

Wider than this, the paper’s authors state that the establishment of mRNA as a safe and effective form of treatment will lead to other forms of RNA therapies coming to the fore, with protein replacement, RNA interference, and CRISPR-Cas gene editing being potential options.

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XTalks

MAY 4, 2021

The CRISPR gene editing system consists of the Cas9 enzyme, which serves as molecular scissors to cleave double-stranded DNA, and a guide RNA template targeted to a specific genomic sequence, which allows for precise editing. Epigenetic Editing with CRISPR.

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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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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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The Pharma Data

FEBRUARY 22, 2021

Gene editing investments advance agriculture with improved food quality, higher yield, and improved crop nutrition. Gene editing is the key to better solutions and greater choices for farmers and consumers around the world. Improving diets through increased quality and access.

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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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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].

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Bioassay Gene Editing Marketing Gene Therapy 52

Delveinsight

AUGUST 27, 2020

The BET inhibitor worked by increasing beta-3 tubulin (TUBB3), a protein found in brain metastases. Now, a research team that is led by scientists at Harvard University have used the CRISPR-Cas9 gene-editing technology to engineer human white fat so that it shows the properties of brown fat.

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The Pharma Data

JANUARY 24, 2021

. “We are excited to welcome Chris, a highly regarded drug developer and team builder who brings to Notch great depth of experience and expertise in development of cell therapies, gene editing, and cell engineering spanning discovery through IND,” said David Main , President and Chief Executive Officer of Notch.

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The Pharma Data

JANUARY 10, 2021

EDIT-301 is an experimental, ex vivo gene editing cell medicine in development for the treatment of sickle cell disease. Previously, the Company received Rare Pediatric Disease designation from the FDA for EDIT-301. EDIT-301 is the first experimental medicine in development generated using CRISPR/Cas12a gene editing.

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pharmaphorum

JUNE 17, 2022

It inhibits the nuclear export protein exportin-1 (XPO1), the same target as Karyopharm’s approved cancer therapy Xpovio (selinexor), which is overexpressed on neurons in ALS in response to stress and may contribute to the pathology of the disease. The post Biogen waves bye to $217m ALS partnership with Karyopharm appeared first on.

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XTalks

DECEMBER 13, 2023

The disease occurs due to a point mutation in the hemoglobin beta globin ( HBB ) gene that codes for one of the proteins that make up hemoglobin, the oxygen carrier in red blood cells. Casgevy’s approval by the FDA is momentous: it is the first CRISPR-based gene-editing therapy to be approved in the US.

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