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.

Gene Editing 81

Gene Editing Genome Genomics Gene 81

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.

Gene Editing 40

Gene Editing Genomics Genome Gene 40

pharmaphorum

JUNE 28, 2021

The reductions matched the efficacy of current therapies for ATTR amyloidosis that require chronic dosing such as Alnylam’s Onpattro (patisiran) and Ionis/Akcea’s Tegsedi (inotersen) – both gene-silencing agents which can cost around $450,000 a year. — Eric Topol (@EricTopol) June 26, 2021.

In-Vivo 105

In-Vivo Gene Editing Trials Drugs 105

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.

Gene Editing 75

Gene Editing Genetics Hormones Medicine 75

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.

Gene Editing 94

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.

Gene Editing 52

Gene Editing Genetics Drugs Gene 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.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

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.

Genomics 52

Genomics Genome Gene Editing DNA 52

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.

Gene Editing 100

Gene Editing DNA Cosmetics Life Science 100

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.

Gene Editing 100

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.

Vaccination 105

Vaccination Vaccine Research Immune Response 105

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.

In-Vivo 52

In-Vivo Gene Editing Gene Therapy Gene 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

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

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].

Bioassay 52

Bioassay Gene Editing Marketing Gene Therapy 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.

Gene Editing 59

Gene Editing Production Development Branding 59

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.

Gene Editing 40

Gene Editing Genome Genomics Protein 40

pharmaphorum

JANUARY 29, 2021

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers.

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

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.

Vaccination 53

Vaccination Vaccine Research Trials 53

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

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.

Gene Editing 40

Gene Editing Trials Licensing Licensing 40

Bioengineer

JULY 23, 2021

“For the first time, we identified a crucial interaction between platelets and the tumor via their surface proteins,” Jain said. The team also works with Dr. Gang Bao, a gene editing expert from Rice University. However, the actual mechanism behind this process remains mostly unknown, until now. ” ###.

Engineer 52

Engineer Gene Editing Clinical Trials Clinical Trials 52

pharmaphorum

NOVEMBER 2, 2021

Using chimeric quadromas, common light chains, and recombinant proteins in bsAb production offers solutions to this issue by limiting the options for association, thus promoting H chain heterodimerisation and forcing cognate HL-chain pairing.

Antibody 52

Antibody Immune Response Production Engineer 52

Pharmaceutical Technology

FEBRUARY 13, 2023

Several biotech companies and researchers are now exploring medical devices and gene therapies to address not just common forms of epilepsy, but also rare conditions such as Dravet Syndrome. Meanwhile, others are researching the link between gene variation and different responses to treatments.

Research 264

Research Gene Gene Therapy Genome 264

pharmaphorum

AUGUST 31, 2022

After a recent approval, there are now three gene therapies available on the US market. In recent years, gene therapy has transitioned from a promising idea to a reality for patients, with many of the severe safety issues that emerged in early iterations of the technology being overcome. from 2021 to 2029.

Gene Therapy 98

Gene Therapy Gene Gene Editing DNA 98

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.

Gene Therapy 98

Gene Therapy Gene FDA Approval In-Vivo 98

XTalks

JANUARY 4, 2024

Pfizer has kickstarted the new year with its first-ever gene therapy approval, awarded by Health Canada to the company’s Beqvez (fidanacogene elaparvovec) for the treatment of hemophilia B. Treatment involves replacing the missing FIX clotting protein, traditionally through regular infusions.

Gene Therapy 111

Gene Therapy Gene Gene Editing Sales 111

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.

Protein 52

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.

In-Vivo 162

In-Vivo Genome Genomics Gene Editing 162

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. Epigenetic Editing with CRISPR. pyogenes dCas9.

DNA 98

DNA Gene Gene Silencing Genome 98

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 99

Gene Immune Response Manufacturing Gene Therapy 99

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 treatment used a new version of CRISPR to turn on a backup copy of the dystrophin gene that mysteriously lies dormant in our muscles.

Gene Editing 105

Gene Editing Gene Gene Therapy Scientist 105

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.

Gene Silencing 98

Gene Silencing Sales Gene Editing Clinical Trials 98

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.

Gene Editing 105

Gene Editing Gene Vaccination Vaccine 105

XTalks

SEPTEMBER 4, 2024

ATTR-CM is a severe and often fatal condition caused by the buildup of misfolded transthyretin (TTR) proteins in the heart, leading to heart failure. Vutrisiran works by silencing the gene responsible for producing TTR, reducing both mutant and wild-type TTR proteins.

Trials 52

Trials RNA Protein Gene Editing 52

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. These modifications regulate gene expression without altering the sequence or structure of DNA.

DNA 52

DNA Gene Gene Silencing Genome 52