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.

Protein 52

Protein Gene Genome Genomics 52

Pharmaceutical Technology

FEBRUARY 15, 2023

Innovation S-curve for the pharmaceutical industry CRISPR nuclease is a key innovation area in pharmaceutical development CRISPR, which refers to clustered regularly interspaced short palindromic repeats, are bacteriophage-derived DNA sequences that had previously infected the prokaryote and are found in the genomes of bacteria and archaea.

In-Vivo 162

In-Vivo Genome Genomics Gene Editing 162

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.

Drugs 52

Drugs Gene Editing Genome Genomics 52

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.

Genome 52

Genome Genomics Gene Editing Genetic Engineering 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.

Production 59

Production Engineer Gene Editing Gene 59

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.”

Gene Therapy 40

Gene Therapy Gene Gene Editing In-Vivo 40

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.

Genetics 52

Genetics Medicine Gene Editing In-Vivo 52

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 Analysis .

Drugs 52

Drugs Gene Editing Genome Genomics 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.

Gene 98

Gene Regulation Protein Gene Editing 98

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.

Gene Editing 98

Gene Editing Gene Drugs In-Vivo 98

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.”.

Gene Editing 40

Gene Editing Genome Genomics Protein 40

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.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 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

. “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 Genome Genomics 52

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.

Gene 73

Gene Immune Response Manufacturing Gene Therapy 73

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.

FDA Approval 111

FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

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.

Vaccination 53

Vaccination Vaccine Research Trials 53

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. .

RNA 52

RNA Genetics Gene In-Vitro 52

Drug Discovery World

MARCH 9, 2023

This includes the measurement of plasma kallikrein protein levels and clinical activity as determined by HAE attack rate measures. The post FDA clears in vivo gene editing therapy for trials in US appeared first on Drug Discovery World (DDW).

In-Vivo 52

In-Vivo Gene Editing Gene Trials 52

Drug Discovery World

OCTOBER 16, 2023

Maina Bhaman, Partner at Sofinnova Partners, says: “The UK is an attractive space for drug discovery companies and a prime environment for investment due to its talent, infrastructure, and genomics research. We also recently opened the Scientific Centre of Excellence for Genomic Insight at our Cambridge site.”

Drugs 75

Drugs Life Science Gene Therapy Genome 75

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.

Genome 52

Genome Genomics Gene Editing DNA 52

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. In his work, Ferraro focuses on the links between gene variations and different responses to drugs.

Research 264

Research Gene Gene Therapy Genome 264

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.

Gene Editing 98

Gene Editing Gene DNA Engineer 98

pharmaphorum

JUNE 28, 2021

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . — Eric Topol (@EricTopol) June 26, 2021. . — Eric Topol (@EricTopol) June 26, 2021.

In-Vivo 104

In-Vivo Gene Editing Trials Drugs 104

Drug Discovery World

MAY 2, 2023

The CRISPR-Cas9 system, awarded the Nobel Prize in Chemistry in 2020 for its applications for gene editing, was initially encountered in the bacterial immune system. In an effort to defend themselves against viral infection, bacterial cells capture and copy DNA fragments of bacteriophages into their genome.

Genome 52

Genome Genomics Gene DNA 52

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 Genome Genomics 119

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. While type II (Cas9) is mainly used for gene-editing applications, CRISPR detecting tools primarily rely on type V (Cas12) and type VI (Cas13) collateral activity.

DNA 98

DNA Genome Genomics RNA 98

Drug Discovery World

APRIL 14, 2023

He is being recognised for his revolutionary contributions to developing the first gene-edited cell-based therapy for cancer that involves the genetic re-engineering of a patient’s own T cells to combat their disease, and for demonstrating that adoptive T-cell therapy can induce remission and in some cases cure patients with advanced cancer.

Genetics 52

Genetics Research Medicine Clinical Research 52

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. The success of base editing has led to rapid advances toward commercialisation and clinical application.

Gene Editing 52

Gene Editing Gene Engineer DNA 52

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.

Gene Editing 52

Gene Editing DNA Genome Genomics 52

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.

Gene Therapy 97

Gene Therapy Gene Gene Editing DNA 97

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.

DNA 98

DNA Gene Gene Silencing Genome 98

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.

Medicine 52

Medicine Development Clinical Trials Clinical Trials 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. 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 Genome 52

Drug Discovery World

MAY 30, 2023

At PhoreMost, we have been particularly excited about the emergence of targeted protein degradation (TPD) as a new therapeutic modality. The concept of harnessing the ubiquitin-proteosome system to ‘destroy’ or degrade disease causing proteins has been established for some time.

Research 52

Research Drugs Protein RNA 52

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.

Gene 89

Gene Protein Gene Expression Scientist 89

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