XTalks

SEPTEMBER 9, 2021

The US Food and Drug Administration (FDA) has placed a clinical hold on BioMarin Pharmaceutical’s investigational gene therapy BMN 307 for the rare inherited disease phenylketonuria (PKU) over safety concerns found during preclinical testing. The mice developed the tumors one year after being given BMN 307.

Gene Therapy 105

Gene Therapy Gene Genomics Genome 105

pharmaphorum

SEPTEMBER 6, 2021

BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PKU) on hold while it investigates a safety signal. “We are committed to understand and mitigate any risk of cancer causation.”

Gene Therapy 94

Gene Therapy Gene Genetic Disease DNA 94

Pharmaceutical Technology

FEBRUARY 3, 2023

With scientists fervently developing mRNA vaccines, nucleic acid therapeutics, and viral vector-based gene therapies, clinicians are set to have a growing number of tools available to treat a wide range of conditions, from infectious diseases to genetic disorders and more. So, we set out to solve some of the problems,” she explains.

Genome 244

Genome Genomics Medicine Gene Therapy 244

BioTech 365

SEPTEMBER 21, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Regulator proteins or symphonies of genes: Statistical modeling points way toward unified theory for DNA folding.At

DNA 40

DNA Regulation Protein Gene 40

Roots Analysis

OCTOBER 16, 2023

DNA cloning refers to the process of generating multiple copies of a particular DNA segment. DNA and Gene Cloning involves the isolation of a DNA sequence of any species ( often a gene ) and its insertion into a vector to enable growth without any alteration in the original DNA sequence.

DNA 40

DNA Gene Protein In-Vitro 40

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. It’s a great tool for controlling gene expression.”.

DNA 98

DNA Gene Gene Silencing Genomics 98

BioPharma Reporter

JANUARY 17, 2022

Andelyn Biosciences says the FDA's acceptance of its GMP plasmid DNA drug master file (DMF) enables the CDMO to vertically integrate its clientsâ manufacturing process, condensing timelines for developers to begin manufacturing to just three months.

Gene Therapy 103

Gene Therapy Development DNA Gene 103

Scienmag

JANUARY 11, 2022

Enhancers are regions of DNA that do not code for proteins, but control how genes are expressed. Super-enhancers are clusters of enhancers that together regulate genes with important roles in cell identity. Scientists at St. Their assessment showed the super-enhancer has four […].

Development 80

Development DNA Gene Protein 80

BioPharma Reporter

OCTOBER 22, 2020

The use of CRISPR, the genetic scissors that allow scientists to edit the instruction manual of life, DNA, has drawn massive global attention over the last several years.

Gene Editing 105

Gene Editing DNA Genetics Scientist 105

XTalks

OCTOBER 8, 2024

The 2024 Nobel Prize in Physiology or Medicine has been awarded to American scientists Victor Ambros and Gary Ruvkun for their groundbreaking discovery of microRNA (or miRNA) and its role in post-transcriptional gene regulation. A gene contains instructions within our DNA.

Regulation 52

Regulation Genetics Scientist RNA 52

pharmaphorum

MARCH 10, 2021

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. bluebird bio’s Philip Gregory.

Gene Therapy 59

Gene Therapy Gene Trials Gene Expression 59

Roots Analysis

AUGUST 31, 2023

Overview of Gene Switch The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E. Gene switches are sites on genes where regulatory molecules can bind to trigger transcription process, leading to expression of a particular gene.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

pharmaphorum

OCTOBER 7, 2020

Drs Emmanuelle Charpentier and Jennifer Doudna have won this year’s Nobel Prize for chemistry in recognition of their work on the gene-editing technology CRISPR/Cas9. The technique introduces a break in a specific place within DNA that triggers a self-repair mechanism. — The Nobel Prize (@NobelPrize) October 7, 2020.

Gene Editing 105

Gene Editing Bacteria DNA Gene 105

BioTech 365

APRIL 5, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: DNA methylation from bacteria and microbiome using nanopore technology.Bacterial DNA methylation occurs in diverse sequence contexts and plays important functional roles in cellular defense and gene regulation.

Bacteria 40

Bacteria DNA Gene Regulation 40

XTalks

JANUARY 21, 2025

The tricuspid valve, one of the heart’s four valves, regulates blood flow from the right atrium to the right ventricle, preventing backflow between these chambers. The Shield test provides a non-invasive approach to colorectal cancer screening by analyzing blood for key DNA changes linked to cancer. Photo courtesy of BD.

FDA Approval 105

FDA Approval Manufacturing Development RNA 105

BioPharma Reporter

JANUARY 9, 2023

The collaboration will combine ReCodeâs delivery technology with AskBioâs gene editing and DNA cargoes to develop gene correction therapies for liver and lung diseases.

Gene Editing 52

Gene Editing Gene DNA Development 52

Scienmag

JANUARY 4, 2021

Study suggests the impact of non-coding microRNAs on circadian rhythms is tissue specific and may reveal new insights into disease processes If you’ve ever had a bad case of jet lag, you know how a disruption to your body’s circadian rhythm makes it difficult to function.

DNA 64

DNA Regulation Gene Genetics 64

The Pharma Data

SEPTEMBER 3, 2020

Current scientific techniques are not yet safe or effective enough to be used to create gene-edited babies, an international committee says. The world’s first gene-edited babies were born in China in November 2018. Why is gene-editing babies controversial? The committee was set up in response. What does the report say?

Gene Editing 52

Gene Editing Gene Genomics Genome 52

pharmaphorum

SEPTEMBER 14, 2020

While there may be just under 20,000 confirmed protein coding genes, it turns out that much of the genome outside of these genes is also important in regulating how the genome is controlled. These technologies directly sequence single molecules of DNA in real time, often without the need for amplification.

Genomics 119

Genomics Genome Medicine Gene 119

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 Genomics Genome 52

Worldwide Clinical Trials

JULY 8, 2024

Genetic Factors : Certain genetic mutations, such as those in DNA repair genes, can affect how cells respond to radiation. Individuals with familial cancer syndrome may have inherited mutations that predispose them to cancer development, affecting genes involved in DNA repair and cell cycle regulation.

Hormones 130

Hormones Regulation Clinical Trials Clinical Trials 130

Pharmaceutical Technology

DECEMBER 14, 2022

Bacteria naturally develop resistance to antibiotics over time, through genetic mutation, horizontal gene transfer between bacteria, or because of selective pressure. In the same way that viruses attack human cells and replicate using machinery in the human cell, phages insert their DNA into a bacterial cell through transduction.

Bacteria 130

Bacteria DNA Clinical Trials Clinical Trials 130

Advarra

JUNE 12, 2023

Gene therapy research is exciting and full of promise, but because of the risks involved, it’s also highly regulated, requiring an institutional biosafety committee (IBC) to provide additional oversight and risk assessment. What do the Regulations Say about IBC Review? What Does an IBC Review?

Genetic Engineering 98

Genetic Engineering Gene Therapy Genetics Engineer 98

Pharmaceutical Technology

JUNE 4, 2023

Within the emerging innovation stage, cell therapy for ocular disorders, coronavirus vaccine components, and DNA polymerase compositions are disruptive technologies that are in the early stages of application and should be tracked closely.

Genetics 130

Genetics Gene Expression DNA Antibody 130

pharmaphorum

AUGUST 16, 2022

The US regulator has started a priority review of Lynparza (olaparib) in combination with abiraterone and prednisone or prednisolone as a first-line treatment for metastatic castration-resistant prostate cancer (mCRPC) based on the results of the PROpel trial. months and 16.6 months, respectively.

Hormones 111

Hormones Gene Sales Trials 111

Worldwide Clinical Trials

DECEMBER 12, 2022

In some cases, the gene associated with a patient’s condition may not yet be discovered or included on the test ordered and the interpretation of genetic variants may change over time as new information is uncovered. single gene testing vs comprehensive panel), the reportable range (e.g.,

Clinical Trials 130

Clinical Trials Clinical Trials Genetics Trials 130

XTalks

APRIL 6, 2022

Cancerous cells and tumors release small fragments of DNA into the bloodstream called circulating tumour DNA (ctDNA). The researchers at the Cancer Research UK Cambridge Institute published this data from the LUng cancer CIrculating tumour DNA (LUCID) study in the journal Annals of Oncology.

DNA 105

DNA In-Vitro Doctors Doctor 105

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 Genomics 52

XTalks

MAY 22, 2023

Related: 5 Food Companies Working With Precision Fermentation Technology How Pairwise Leverages CRISPR Technology CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats, is a gene-editing technique that can be used to alter the DNA of cells to enhance certain characteristics or reduce less desirable ones.

Branding 98

Branding Gene Editing DNA Production 98

XTalks

DECEMBER 1, 2020

Circulating DNA. The test is based on the identification of cancer-associated methylation signatures on DNA shed from tumor cells in the blood. The addition of methyl groups to DNA bases — namely cytosine and guanine in so-called ‘CpG islands’ — regulates the expression of genes.

DNA 64

DNA Genome Genomics Gene 64

XTalks

MARCH 13, 2024

There are also studies examining gene therapy-based techniques to repair or replace genetic defects contributing to this disease. The Glaucoma Foundation has funded studies focusing on two specific areas within gene therapy. Secondly, other funded research is exploring differential gene expression regulated by DNA methylation.

Gene Therapy 111

Gene Therapy Gene Life Science DNA 111

Scienmag

JANUARY 11, 2021

These biomarkers are epigenetic, meaning they involve changes to molecular factors that regulate genome activity such as gene expression independent of DNA sequence, and can be passed down to future generations. In a […].

Gene Expression 41

Gene Expression DNA Genome Genomics 41

Pharmaceutical Technology

JUNE 30, 2022

These patterns hint at the factors playing a role in generating mutations, be it environmental risks such as UV light or smoking or DNA repair mechanism defects that cause internal mutations, giving a very individual picture of each cancer from a genomic point of view, says Degasperi. Still, some key questions remain unanswered.

Genome Project 130

Genome Project Genomics Genome DNA 130

The Pharma Data

AUGUST 28, 2020

FoundationOne Liquid CDx analyses more than 300 cancer-related genes and multiple genomic signatures to help inform treatment decisions for all solid tumour cancers.

Genome 52

Genome Genomics Gene Regulation 52

WCG Clinical

JUNE 24, 2024

Genetic manipulation has an interesting history of self-regulation in the United States (US). In 1953, Watson and Crick formulated the current description of DNA as a double helix 1 and soon thereafter experiments with restriction endonucleases gave scientists the ability to modify and manipulate DNA 2.

DNA 52

DNA Regulation Scientist Research 52

pharmaphorum

OCTOBER 19, 2022

The dark genome – a loose term that covers non-coding regions of the genome that are capable of regulating the expression of genes, previously rather inaccurately referred to as junk DNA – is increasingly being explored by biopharma companies for new drug targets. Dr Danuta Jeziorska.

Genomics 59

Genomics Genome Gene Gene Expression 59

The Pharma Data

NOVEMBER 24, 2020

BRCA 1/2 or ATM gene mutation must be confirmed before Lynparza treatment is initiated. 2 Germline and somatic mutations in HRR genes, which include BRCA1/2 and ATM, are associated with potentially more aggressive prostate cancers and poor prognosis, demonstrating the importance of biomarker testing. MISSISSAUGA, ON , Nov.

Gene 52

Gene Hormones DNA Medicine 52