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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Gene Editing Gene DNA Engineer 98

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 Genomics Genome 52

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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In-Vivo Gene Editing Trials Gene 52

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 Genomics Genome 52

XTalks

MAY 4, 2021

The tool could also prove to be safer than conventional CRISPR-based gene therapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. Epigenetic Editing with CRISPR. Nearly one third of human genes lack CGIs, which would limit the use of the tool.

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DNA Gene Gene Silencing Genomics 98

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

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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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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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. This enhances sustainability and helps us reduce the environmental impact of agriculture.”.

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Gene Editing Genomics Genome Protein 40

The Pharma Data

JANUARY 10, 2021

Nasdaq: EDIT), a leading genome editing company, today announced the U.S. Food and Drug Administration (FDA) has cleared the initiation of the safety phase of the Company’s EDIT-301 clinical trial, and the Company can begin dosing patients. Enrolled patients will receive a single administration of EDIT-301.

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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.

Engineer 52

Engineer Gene Editing Genomics Genome 52

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. In 2021, the FDA granted approval to San Francisco-based Excision BioTherapeutics to start trials assessing CRISPR gene editing as a potential treatment for HIV.

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FDA Approval Gene Therapy Gene Clinical Trials 111

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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Gene Immune Response Manufacturing Gene Therapy 99

Delveinsight

AUGUST 27, 2020

Freenome’s diagnostic searches for direct indication of tumors and signs of the immune system’s response, integrating genomics, transcriptomics, methylomics, and proteomics technologies. The BET inhibitor worked by increasing beta-3 tubulin (TUBB3), a protein found in brain metastases.

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Vaccine Vaccination Research Trials 53

XTalks

MAY 4, 2021

The tool could also prove to be safer than conventional CRISPR-based gene therapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. Epigenome Editing with CRISPR. Nearly one third of human genes lack CGIs, which would limit the use of the tool.

DNA 52

DNA Gene Gene Silencing Genomics 52

XTalks

MARCH 28, 2024

Gene Editing and CAR T-Cell Therapy Genetic engineering technologies, such as CRISPR-Cas9, are providing newer potential avenues for cancer treatment. For example, gene editing can possibly be used to correct cancer-causing mutations or enhance the effectiveness of chimeric antigen receptor (CAR) T-cell therapy.

Gene Editing 59

Gene Editing Life Science Clinical Trials Clinical Trials 59

The Pharma Data

JANUARY 24, 2021

It includes adults with confirmed COVID-19 on supplemental oxygen or mechanical ventilation with abnormal levels of at least two serum biomarkers, C reactive protein, D dimer, lactate dehydrogenase, or ferritin. CYAD-101 is the company’s allogeneic NKG2D-receptor and T cell receptor inhibitory molecule-based, non-gene edited CAR-T candidate.

Trials 52

Trials Allergies In-Vitro Antibody 52

XTalks

DECEMBER 24, 2020

From isolating SARS-CoV-2 in early January to sequencing its genome shortly thereafter and having a prototype vaccine against it within days, scientific process and progress have held steadfast throughout the pandemic. Most antibody drugs and vaccines have been developed to target parts of the spike protein. Innovations.

Life Science 111

Life Science Vaccine Vaccination Gene 111

XTalks

FEBRUARY 11, 2021

The Human Genome Project recently marked 20 years since the publication of the first full sets of human genomic sequences, an endeavor that spanned well over a decade. Today, new next-generation sequencing technologies allow for the sequencing of complex genomes within just a day or two. Martha Chase: For the Books.

Genetics 119

Genetics DNA Genomics Genome 119

XTalks

SEPTEMBER 21, 2020

It helps explain why women with the disease survive longer with less severe symptoms than men during the early stages of the Alzheimer’s, despite having the same levels of toxic amyloid beta and tau proteins in their brains. The gene, called KDM6A , is a histone demethylase that is believed to function as a tumor suppressor.

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Gene Protein Gene Expression Scientist 89

XTalks

JANUARY 6, 2025

RNA-Based Therapeutics RNA molecules have emerged as promising therapeutic agents due to their ability to precisely target undruggable proteins or molecules, often with minimal side effects. Additionally, CRISPR genome-wide screening holds great potential for identifying key disease-associated genes and uncovering novel therapeutic targets.

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