pharmaphorum

OCTOBER 7, 2020

Originally discovered in bacteria as a defense mechanism against pathogens, CRISP/Cas9 has made it possible to make exquisitely detailed and precise alterations to DNA sequences on demand, and as a tool for molecular biology has already transformed research into diseases and drug discovery. What started as a curiosity?

Gene Editing 105

Gene Editing Bacteria DNA Gene 105

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

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? How is IBC Membership Composed?

Genetic Engineering 98

Genetic Engineering Gene Therapy Genetics Engineer 98

Scienmag

APRIL 15, 2021

Epigenetic regulator HPI1a drives de novo genome reorganization in early Drosophila embryos Credit: MPI of Immunobiology and Epigenetics, F. Zenk The DNA molecule is not naked in the nucleus. This […].

Genomics 56

Genomics Genome DNA Genetics 56

pharmaphorum

AUGUST 10, 2021

Europe market has proved so hostile to gene therapies when it comes to pricing and reimbursement for gene therapies that bluebird bio has decided to quit the market altogether, according to Andrew Obenshain, president of its severe genetic diseases unit. market and will wind down in Europe. — Brad Loncar (@bradloncar) August 9, 2021.

Gene Therapy 135

Gene Therapy Gene Marketing Genetic Disease 135

Worldwide Clinical Trials

JUNE 21, 2024

Although there are a range of efficacies across different tumor types that require a greater understanding, the use of circulating tumor DNA profiles holds promise for personalized treatment strategies that better target the drug for the patient, potentially revolutionizing how ADCs are deployed in clinical practice.

Antibody 162

Antibody Clinical Trials Clinical Trials In-Vitro 162

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

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

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

FEBRUARY 4, 2021

Bernard, executive vice president for research and chief research officer at Vanderbilt University Medical Center, shared his experience in helping set up a DNA and clinical data resource called BioVU. The project began in 2004, in the early days of biobanking for DNA, says Dr. Bernard, and has grown tremendously since.

Research 98

Research Biobanking Drugs Genome 98

Scienmag

DECEMBER 16, 2020

New research from the Whitehead Institute suggests that the products of transcription — RNA molecules — regulate their own production through a feedback loop Credit: Jon Henninger/Whitehead Institute At any given moment in the human body, in about 30 trillion cells, DNA is being “read” into molecules of messenger RNA, the intermediary (..)

RNA 52

RNA DNA Regulation Production 52

XTalks

DECEMBER 28, 2023

XTALKS WEBINAR: Impacts of the New IVD Regulation (IVDR) for Manufacturers and Users Live and On-Demand: Tuesday, January 9, 2024, at 10am EST (4pm CET/EU-Central) Register for this free webinar to learn how the new In Vitro Diagnostic Regulation (IVDR) impacts manufacturers and users of diagnostic devices.

Trials 97

Trials Clinical Trials Clinical Trials Antibody 97

pharmaphorum

SEPTEMBER 14, 2020

Last week geneticist Dr Charles Steward shared with us his experiences of searching for a genetic cause for his children’s rare neurological diseases. 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.

Genomics 119

Genomics Genome Medicine Gene 119

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

Scienmag

NOVEMBER 16, 2020

Wnt-regulated lncRNA discovery enhanced by in vivo identification and CRISPRi functional validation. doi:10.1186/s13073-020-00788-5… SINGAPORE, 3 Nov 2020 – Long RNA molecules carrying DNA codes that […]. Harmston, N., Glaser, T.L. Genome Med 12, 89 (2020).

In-Vivo 53

In-Vivo RNA DNA Genome 53

The Pharma Data

AUGUST 28, 2020

US regulators have approved Roche’s new pan-tumour liquid biopsy test for patients with solid tumours, offering a potentially more convenient option and an alternative for patients unable to have tissue biopsy.

Genome 52

Genome Genomics Gene Regulation 52

pharmaphorum

SEPTEMBER 6, 2021

The biotech said that the US regulator ordered the pause on the study after liver tumours were seen in mice given the therapy in preclinical testing. PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down phenylalanine, an amino acid that is commonly found in many foods.

Gene Therapy 94

Gene Therapy Gene Genetic Disease DNA 94

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

WCG Clinical

AUGUST 27, 2024

1 This is an important requirement, as the NIH Guidelines were originally created partly in response to public concerns surrounding recombinant DNA and genetic engineering. Many of those original concerns of the 1970s and 1980s surrounding genetic engineering have lessened over time.

Genetic Engineering 52

Genetic Engineering Engineer Compliance Genetics 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. Shares in bluebird ticked up following the announcement.

Gene Therapy 59

Gene Therapy Gene Trials Gene Expression 59

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

XTalks

MARCH 13, 2024

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

Gene Therapy 111

Gene Therapy Gene Life Science DNA 111

FDA Law Blog

JANUARY 5, 2023

Claud — As we turn into the New Year, we offer a few items of interest in digital and telehealth regulation, enforcement, and compliance that may provide some helpful guideposts for stakeholders. This was a topic at the December Food and Drug Law Institute conference on Current Developments in Digital Health Technology and Regulation.

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Regulation Production Drug Delivery Marketing 59

Pharmaceutical Technology

APRIL 24, 2023

In an email to this news service, João Conde, PhD, professor at NOVA Medical School, Universidade NOVA de Lisboa, said advances like the mRNA vaccines serve as a testament to the breakthroughs made by science over decades of research at the junction of genetics and nanomedicine. These could feature in the next four to five years.

Drug Delivery 147

Drug Delivery Vaccine Vaccination Research 147

The Pharma Data

NOVEMBER 27, 2020

Nasdaq:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, announced today that the U.S. With this approval, Imcivree becomes the first-ever FDA approved therapy for these rare genetic diseases of obesity. BOSTON, Nov.

FDA Approval 52

FDA Approval Genetic Disease Genetics Clinical Trials 52

The Pharma Data

OCTOBER 18, 2020

(NASDAQ:CDXC) today highlighted a new study published in The European Molecular Biology Organization Journal looking at the effect of nicotinamide riboside (NR) on maintaining telomeres, the protective regions at the end of DNA strands. Telomeres are “caps” at the end of chromosomes that protect DNA from getting worn away as cells replicate.

DNA 52

DNA Scientist Genetic Disease Research 52

XTalks

APRIL 5, 2021

Specific skills are required to succeed in this job; these include advanced writing skills for scientific literature, developing software tools, working on genome sequencing projects, and designing DNA sequencing libraries. Having a strong background in biochemistry or genetics is necessary for the problem-solving element of the job.

Bioinformatics 59

Bioinformatics Engineer Scientist Genome 59

Cloudbyz

NOVEMBER 3, 2021

They are also making a profit by offering additional services related to sample processing, such as DNA extraction and cutting sections of genetic material. If the trend progresses with no market hindrances or opposing regulations on a global scale, sourcing Human Biosamples may become more equitable in the future.

Biobanking 52

Biobanking Clinical Research Research Contamination 52

XTalks

SEPTEMBER 30, 2020

The research shows that the activity of the immune messenger type 1 interferon (IFN) protein is diminished, either by genetic mutations or an autoimmune attack by neutralizing antibodies against it, in a subset of COVID-19 patients. Casanova’s lab is now searching for the genetic driver behind the autoantibodies.

Antibody 135

Antibody Genetics Research In-Vitro 135

Pharmaceutical Technology

JUNE 4, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: Transcription factors for genetically modified cells. However, not all innovations are equal and nor do they follow a constant upward trend.

Genetics 130

Genetics Gene Expression DNA Antibody 130

pharmaphorum

JULY 20, 2022

It has suspected for many years that some diseases may be linked to non-coding or ‘junk’ DNA, but the mechanism behind the pathology hasn’t been worked out. Junk DNA is a term used to describe the 97% of the genetic sequence in human cells found between the 3% coding for our 20,000 genes, once thought to be inert.

DNA 116

DNA Scientist Genome Genomics 116

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

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

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. The genomic medicine journey. Pragmatic genomics.

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

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

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.

Genomics 244

Genomics Genome Medicine Gene Therapy 244

XTalks

OCTOBER 1, 2020

Sooter has also found success in industry, leading and bringing together the work of different teams of researchers at NeuBase to help drive the company’s goal of developing cures and treatments for genetic diseases, including Huntington’s Disease. She says, “It’s been a lot of fun; it’s a decision I’m glad I made.”.

Scientist 52

Scientist In-Vitro Research Life Science 52