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).

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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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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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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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Pharmaceutical Technology

MAY 25, 2023

ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. The technology platforms include the Life Edit gene editing platform, an RNA, cell, protein, vector engineering and induced pluripotent stem cells (iPSCs) platform.

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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

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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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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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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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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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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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

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. The two patients treated with 0.45mg/kg had a time-averaged blood PCSK9 protein reduction of 59% and 84%.

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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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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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Camargo

JULY 27, 2021

Gene therapy is a new therapeutic approach in which genes are used to treat or prevent diseases. It is a comprehensive term which encompasses a large variety of therapy products including viral and bacterial vectors, plasmid DNA, human gene editing technology, and patient-specific cellular gene therapy.

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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 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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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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Drug Discovery World

AUGUST 2, 2022

It’s estimated that one in 250 people worldwide are affected by genetic cardiomyopathies, with a 50:50 risk they will pass their faulty genes on to each of their children. . CureHeart is using the gene editing technology CRISPR to deploy a technique called base and prime editing in the heart for the first time.

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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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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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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

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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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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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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Drug Discovery World

NOVEMBER 17, 2023

The breaking news this week was certainly the first regulatory approval for Casgevy, not only the first gene therapy approved for sickle cell disease, but also the first therapy to use CRISPR gene editing. However, there were a number of other breakthroughs announced in the cell and gene therapy field.

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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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Drug Discovery World

MARCH 22, 2023

“Each of these two regulatory genes has been implicated in restricting T cell inflammatory responses, but we found that disrupting them together produced much greater anticancer effects than disrupting them individually,” Mai said. “By

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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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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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Drug Discovery World

FEBRUARY 9, 2024

Primary hepatocytes represent the gold standard in vitro cell model for functionality, however, when isolated from liver tissue, primary cells maintain expression of key proteins for only a short period. However, hepatoma lines may lack the key proteins essential for modelling. Are there any ethical boundaries behind the use of iPSCs?

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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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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

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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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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Gene Editing Gene Vaccination Vaccine 101