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

Scienmag

MAY 7, 2021

The two meters of -stretched- DNA contained in human cells are continuously twisting and untwisting to give access to genetic information: when a gene is expressed to generate a protein, the two strands of DNA are separated to give access to all the machinery necessary for this expression, resulting in an excessive accumulation of coiling […]. (..)

DNA 62

DNA Regulation Genome Genomics 62

Pharmaceutical Technology

JUNE 13, 2023

Ziftomenib targets the menin-KMT2a protein-protein interaction that may occur in patients with KMT2ar or NPM1m genetic alterations. The aberrant protein complex is essential for regulating leukaemia-promoting gene expression. Thus, targeting menin may be an effective strategy for patients with high unmet needs.

Gene Expression 130

Gene Expression Protein Sales Genetics 130

Scienmag

OCTOBER 27, 2021

The rapid scientific advancements that followed the mapping of the human genome have revealed just how staggeringly complex the world of genetics is. We now know that proteins are not just the products of genes, but that they also interact with genes, influencing and regulating the rhythm of their expression.

Gene 72

Gene Genomics Genome Genetics 72

pharmaphorum

SEPTEMBER 14, 2020

Merck & Co has signed a multi-billion cancer drug development deal, which will see it investing $1 billion in partner Seattle Genetics. The drug zeroes in on cancer cells expressing a protein known as LIV-1 and is in phase 1 development for breast cancer and other solid tumours. billion equity investment in 5.0

Genetics 52

Genetics Development Drugs Protein 52

Outsourcing Pharma

JUNE 7, 2023

Duchenne Muscular Dystrophy (DMD) is a genetic disorder that primarily affects males, caused by a mutation in the gene that encodes for a protein called dystrophin. This protein is crucial for the normal functioning of muscles, and its absence leads to progressive muscle weakness and wasting.

Protein 82

Protein Genetics Gene Research 82

Scienmag

SEPTEMBER 22, 2020

Brazilian researchers observed that in uninfected adipocytes, the hormone irisin altered the expression of genes that regulate ACE-2, which encodes a protein to which the virus binds in order to invade human cells.

Hormones 97

Hormones Gene Protein Regulation 97

The Pharma Data

MARCH 23, 2022

Polymerase is a viral protein that directs how Ebola virus replicates its genome as it infects new hosts. The researchers discovered that Ebola virus polymerase hijacks a cellular protein called GSPT1. To treat Ebola virus infections, researchers are taking a close look at a key piece of the virus: polymerase.

Protein 52

Protein Genome Genomics Research 52

Medical Xpress

MARCH 21, 2025

A team of researchers has identified a mechanism that regulates the production of two different proteins from the same gene. carried out their study with the nematode C. elegans, a small worm widely used in biological research as an animal model.

Regulation 40

Regulation Protein Gene Research 40

The Pharma Data

DECEMBER 3, 2020

New Tau Protein is Abnormal in Very Early Alzheimer’s Disease. Investigators with the University of Gothenburg identified new forms of tau protein that become abnormal in the very early stages of Alzheimer’s disease, even before the development of cognitive problems. “The Every week there are numerous scientific studies published.

Protein 52

Protein Research Gene Regulation 52

pharmaphorum

SEPTEMBER 14, 2020

AstraZeneca has resumed UK trials for its coronavirus vaccine, after the country’s medicines regulator gave the all-clear following a safety scare. After vaccination, the surface spike protein is produced, priming the immune system to attack the SARS-CoV-2 virus if it later infects the body.

Vaccination 104

Vaccination Vaccine Trials Protein 104

Scienmag

APRIL 15, 2021

Epigenetic regulator HPI1a drives de novo genome reorganization in early Drosophila embryos Credit: MPI of Immunobiology and Epigenetics, F. Instead, it is folded in a very organized way by the help of different proteins to establish a unique spatial organization of the genetic information. This […].

Genome 56

Genome Genomics DNA Genetics 56

pharmaphorum

AUGUST 23, 2022

France’s BrainVectis, a subsidiary of Bayer’s Asklepios BioPharma (AskBio) unit, has been given the green light by regulators in France to start dosing patients with its gene therapy candidate for devastating neurodegenerative disorder Huntington’s disease.

Gene Therapy 105

Gene Therapy Gene Trials Protein 105

Scienmag

AUGUST 4, 2020

Led by Boyce Thompson Institute’s Andrew Nelson, four partners will identify RNA modifications and develop resources that may lead to hardier crops Credit: Photo credit: Anna Nelson Dittrich ITHACA, NY, August 4, 2020 — RNA perform a variety of functions in cells, helping with everything from regulating genes to building proteins.

RNA 74

RNA Bioinformatics Protein Gene 74

Pharmaceutical Technology

JANUARY 22, 2023

T3 is a major hormone in the blood that regulates thyroid levels. Elsewhere, Albireo Pharma is aiming to file for supplemental NDA to the FDA for its drug Bylvay (odevixibat), which could be the second treatment for Alagille syndrome, a rare genetic disorder caused by a JAG1 gene mutation.

Drugs 317

Drugs Gene Hormones Clinical Trials 317

The Pharma Data

JUNE 14, 2023

Scientists discover small RNA that regulates bacterial infection People with weakened immune systems are at constant risk of infection. Using genetic sequencing technologies, Whiteley and Cao measured the levels of all types of mRNA present in the bacteria. Now, we know that oxygen levels are regulating this transition.”

RNA 40

RNA Regulation Scientist Bacteria 40

pharmaphorum

FEBRUARY 5, 2021

A spokesperson for the MHRA, which has proved to be faster in its decision making than the European system, said the regulator is working with Janssen to complete a rolling review process. “We J&J’s vaccine works by using an adenovirus as vector to deliver the genetic code of the Spike protein found on the coronavirus to the body.

Vaccination 84

Vaccination Vaccine Protein Medicine 84

pharmaphorum

SEPTEMBER 17, 2020

The vaccine is based on a weakened virus that causes cold symptoms in chimpanzee, which causes an immune respone with genetic material coding for the spike protein found on the surface of the coronavirus.

Vaccination 105

Vaccination Vaccine Trials Medicine 105

BioTech 365

FEBRUARY 25, 2021

It transfers genetic information, translates it into proteins or supports gene regulation. … Continue reading →

RNA 40

RNA Genetics Protein Gene 40

pharmaphorum

DECEMBER 1, 2020

Pfizer and BioNTech have filed their COVID-19 vaccine with the European medicines regulator, paving the way for a potential approval before the end of the year. They work by using a short length of genetic code that makes the body produce the “Spike” protein seen on the surface of the SARS-CoV-2 coronavirus.

Vaccination 88

Vaccination Vaccine RNA Clinical Trials 88

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.

Genome 119

Genome Genomics Medicine Gene 119

The Pharma Data

SEPTEMBER 14, 2020

On September 6 the standard review process triggered a voluntary pause to vaccination across all global trials to allow review of safety data by independent committees, and international regulators, after one trial volunteer experienced an unexplained illness. Source link.

Vaccination 52

Vaccination Vaccine Trials Immune Response 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

XTalks

APRIL 3, 2024

The RBCs in circulation are marked by proteins in the complement system for removal by the spleen and liver. AstraZeneca is also venturing into cell therapy and genetic medicine. The complication occurs in these patients after the use of C5 inhibitors because C5 inhibition enables RBCs to survive and circulate.

FDA Approval 102

FDA Approval Sales Trials Development 102

The Pharma Data

NOVEMBER 22, 2020

As the Pfizer and BioNTech COVID-19 vaccine goes to regulators and the Moderna vaccine approaches the end of its Phase III trials, AstraZeneca and the University of Oxford announced high-level results from an interim analysis of their COVID-19 vaccine, AZD1222. It contains the genetic materials of the spike protein.

Vaccination 52

Vaccination Vaccine Protein Genetics 52

XTalks

JANUARY 26, 2023

Diagnosis is confirmed by a differential complete blood count and genetic testing. Treatment selection is determined by age, disease severity, comorbidities and the presence or absence of certain genetic mutations. As with many cancers, CLL and SLL are more difficult to treat in the relapse/refractory setting.

FDA Approval 97

FDA Approval Life Science Clinical Trials Clinical Trials 97

XTalks

MAY 12, 2023

Rett syndrome is a rare genetic disorder that predominantly affects girls and leads to severe physical and cognitive impairments. The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. Dr. Bishop: Rett syndrome is a genetic disorder caused by a mutation in the MECP2 gene.

FDA Approval 97

FDA Approval Genetics Trials Drugs 97

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

XTalks

APRIL 6, 2022

Register for this webinar to get an overview of the role of long non-protein coding RNAs (lncRNAs) in gene regulations. Other MRD blood tests that monitor ctDNA include Signatera™ , developed by US genetic testing and diagnostics company Natera. Live and On-Demand: Thursday, April 21, 2022, at 11am EDT (4pm BST/UK).

DNA 105

DNA In-Vitro Doctors Doctor 105

pharmaphorum

AUGUST 7, 2020

LRRK2 regulates lysosomes, which play a vital role in cell function by breaking down excess or worn-out cell parts, and can also destroy invading viruses and bacteria. Inhibition of LRRK2 activity may slow the progression of Parkinson’s disease in patients with and without known genetic risks based on restoration of lysosomal function.

Bacteria 69

Bacteria Marketing Licensing Licensing 69

XTalks

AUGUST 29, 2022

In addition, following different treatment regimens, cancer cells confer genetic mutations that make these cells even more challenging for the body to recognize. Cytokines are signaling proteins produced by immune cells. They regulate the maturation and growth of immune cells and play an important role in immune mediation.

Clinical Trials 95

Clinical Trials Clinical Trials Trials Drugs 95

The Pharma Data

AUGUST 13, 2020

Mutations in the DMD gene can lead to an absence of the muscle cell protein dystrophin. The drug was trialled in two studies of a total of 32 male participants with genetically confirmed DMD. In one of these studies of 16 patients, Viltepso generated an average increase in dystrophin levels from 0.6% of normal at baseline to 5.9%

FDA Approval 52

FDA Approval Genetics Protein Gene 52

The Pharma Data

MARCH 18, 2021

The UK regulator confirmed that the benefits of the vaccine in preventing COVID-19 far outweigh the risks, and people should continue to get vaccinated when asked to do so. We trust that, after the regulators’ careful decisions, vaccinations can once again resume across Europe.”.

Vaccination 52

Vaccination Vaccine Regulation Protein 52

pharmaphorum

SEPTEMBER 21, 2020

A group of rare genetic disorders, FCS affects between 55 and 110 people in England and causes very high levels of triglyceride fat in the blood. The drug works by reducing the production of ApoC-III, a protein that regulates plasma triglycerides.

Drugs 80

Drugs RNA Medicine Genetics 80

The Pharma Data

JULY 6, 2021

PGRN is a key regulator of immune activity in the brain with genetic links to multiple neurodegenerative disorders, making it one of the most attractive genetically validated targets for the development of new immuno-neurology treatments. ” Arnon Rosenthal, Ph.D.,

Genetics 52

Genetics Gene Antibody Development 52

XTalks

AUGUST 6, 2020

TSC is a rare genetic disease that affects approximately 1 in 6,000 people. The disease is caused by mutations on the TSC1 and TSC2 genes, which produce the proteins hamartin and tuberin, respectively.

FDA Approval 64

FDA Approval Protein Containment Genetic Disease 64

XTalks

JANUARY 3, 2025

FCS is a rare genetic disorder that prevents the body from properly breaking down triglycerides (a type of fat in the blood), leading to dangerously high levels. Tryngolza works by targeting a protein in the liver, apoC-III, which regulates triglyceride metabolism.

FDA Approval 59

FDA Approval Drugs Clinical Trials Clinical Trials 59