Medical Xpress

MARCH 21, 2023

An international study led by the Molecular Physiology Laboratory at the UPF Department of Medicine and Life Sciences (MELIS) identifies new genes that modulate the toxicity of the protein β-amyloid, responsible for causing Alzheimer's disease.

Protein 98

Protein Gene Life Science Research 98

XTalks

MARCH 28, 2024

Duvyzat, a histone deacetylase (HDAC) inhibitor that works to reduce inflammation and muscle loss, is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. Progressive muscle weakness in the disease is caused by genetic mutations in the dystrophin gene that lead to a lack of functional dystrophin protein.

FDA Approval 105

FDA Approval Genetics Gene Therapy Gene 105

Scienmag

MAY 7, 2021

Credit: UPMC PITTSBURGH, May 7, 2021 – In a paper published today in Nature Communications, an international group of collaborators led by researchers at UPMC Children’s Hospital of Pittsburgh have identified a genetic cause of a rare neurological disorder marked by developmental delay and loss of coordination, or ataxia.

Genetics 95

Genetics Genetic Disease RNA Protein 95

Scienmag

SEPTEMBER 7, 2020

Credit: Dr Jie Zheng An innovative genetic study of blood protein levels, led by researchers in the MRC Integrative Epidemiology Unit (MRC-IEU) at the University of Bristol, has demonstrated how genetic data can be used to support drug target prioritisation by identifying the causal effects of proteins on diseases.

Genetics 56

Genetics Protein Drugs Development 56

Medical Xpress

DECEMBER 12, 2022

An international research team led by Dr. 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 (..)

Gene Editing 75

Gene Editing Genetics Hormones Medicine 75

Drug Discovery World

APRIL 3, 2024

In the last few years, immunotherapies like immune checkpoint inhibitors, vaccines and cell and gene therapies have revolutionised the treatment of cancer, and targeted drugs like monoclonal antibodies (mAbs), poly-ADP ribose polymerase (PARP) inhibitors and targeted protein degraders are also playing an important role.

Research 52

Research Drugs Gene Therapy Genetics 52

Drug Discovery World

JULY 24, 2023

The Defense Advanced Research Projects Agency (DARPA) has announced that Ginkgo Bioworks, which is building a platform for cell programming and biosecurity, has been awarded a four-year contract worth up to $18 million to reimagine how to manufacture complex therapeutic proteins.

Protein 52

Protein Manufacturing Gene Sequencing Production 52

Medical Xpress

APRIL 5, 2023

A study by Indiana University School of Medicine researchers sheds new light on the development and treatment of a rare form of muscular dystrophy. The study's findings were recently published in Nature Communications.

Protein 75

Protein Research Medicine Development 75

Drug Discovery World

AUGUST 16, 2023

A new study has shed light on the genetic messages encoded by genes within ‘triple negative’ breast cancers (TNBC), and shows they could predict response to chemotherapy. They suggest it would be better to use biopsies taken from secondary tumours as the cancers can evolve between first diagnosis and recurrence after initial therapy.

Genetics 52

Genetics Gene Hormones RNA 52

Drug Discovery World

MARCH 21, 2023

In a step forward for genetic engineering and synthetic biology, US researchers have modified E coli bacteria to be immune to infection by all natural viruses tested so far. Genetic engineering The findings build on earlier efforts by genetic engineers, which involved genetically reprogramming E.

Engineer 52

Engineer Genetic Engineering Scientist Bacteria 52

Scienmag

FEBRUARY 26, 2021

Researchers from the Andalusian Centre for Molecular Biology and Regenerative Medicine (CABIMER), in collaboration with the Swiss Institute for Experimental Cancer Research (ISREC) have studied the mechanisms behind the higher tendency of people with Mulibrey syndrome to develop tumours.

Protein 40

Protein Genetics Medicine Research 40

Drug Discovery World

SEPTEMBER 4, 2023

In the third and final part of this series of articles focused on plant-based medicines, DDW’s Megan Thomas evaluates therapeutic breakthroughs using plant-based products in drug discovery. In this study, researchers discovered how EGCG breaks apart tangles of the protein tau, a ‘hallmark’ of Alzheimer’s disease.

Medicine 59

Medicine Protein Containment Drugs 59

Medical Xpress

FEBRUARY 7, 2023

University of Saskatchewan (USask) College of Medicine graduate student Ananna Arna dedicated a research project to examine how genetics and DNA replication play a role in leukemia development. In 2022, an estimated 7,000 Canadians were diagnosed with leukemia, a term used to define cancer of the blood cells.

DNA 74

DNA Protein Genetics Medicine 74

Drug Discovery World

SEPTEMBER 27, 2023

Founder Dr Moshe Szyf Founder shares the potential of harnessing precision medicine. Embarking on a new era of medicine The advent of genomics has ushered in the era of personalised medicine, enabling us to analyse the genetic makeup of individuals with unprecedented accuracy.

DNA 115

DNA Genetics Gene Medicine 115

FDA Law Blog

DECEMBER 13, 2023

Sasinowski — On December 12, 2023, FDA announced the creation of a new advisory committee specifically for treatments for genetic metabolic diseases, the Genetic Metabolic Diseases Advisory Committee, or “GeMDAC.” There are hundreds of known genetic metabolic diseases, most of which are rare and carry significant morbidity.

Genetics 59

Genetics Production Development Drugs 59

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. Every week there are numerous scientific studies published.

Protein 52

Protein Research Regulation Gene 52

Pharmaceutical Technology

APRIL 26, 2023

MiNA Therapeutics has entered into a research collaboration and option licensing agreement with BioMarin Pharmaceutical to speed up the development of therapeutic ribonucleic acid activation (RNAa) candidates to treat rare genetic diseases. The new deal excludes oncology and other therapeutic areas outside the scope of genetic disease.

Development 130

Development Genetic Disease Genetics Gene Therapy 130

Drug Discovery World

JULY 5, 2023

Global Cancer Segment Market Manager at Agilent Technologies, recently sat down with DDW Editor Reece Armstrong to tell us about the developments in the ‘golden age’ of cancer research. RA: What is the current state of the cancer research industry? Mark Garner, PhD., MG: I am responsible for Agilent’s cancer segment.

Research 98

Research Protein Scientist Immune Response 98

Scienmag

FEBRUARY 3, 2021

Scientists illuminate the protein’s role in rare genetic diseases often diagnosed during infancy or childhood Scientists at Scripps Research have clarified the workings of a mysterious protein called G?o, o, which is one of the most abundant proteins in the brain and, when mutated, causes severe movement disorders.

Protein 42

Protein Genetic Disease Genetics Scientist 42

Pharmaceutical Technology

JULY 5, 2022

Researchers at the UNC School of Medicine have discovered that hepatitis A virus (HAV) replication needs particular interactions between the human protein ZCCHC14 and TENT4 poly(A) polymerases, a group of enzymes. In addition, the scientists later found that the HAV needs TENT4A/B for its replication.

Protein 130

Protein RNA Genome Genomics 130

Scienmag

MAY 12, 2021

Mutations can disrupt protein binding through a “burr effect” thus interfering with the regulation of cell growth Credit: Kümmel team/Oeckinghaus team Tuberous Sclerosis Complex (TSC) affects between one and two of every 10,000 new-born babies.

Genetic Disease 94

Genetic Disease Research Genetics Protein 94

Scienmag

JULY 2, 2021

Studies using human cell lines and tumors grown in mice provide early evidence that inhibiting RNA-binding proteins, a previously overlooked family of molecules, might provide a new approach for treating some cancers Credit: UC San Diego Health Sciences In cancer research, it’s a common goal to find something about cancer cells — some sort (..)

RNA 87

RNA Protein Drugs Research 87

Medical Xpress

JANUARY 2, 2023

Studying mice, researchers at Washington University School of Medicine in St. Louis have found that proteins made by stem cells that regenerate the cornea may be new targets for treating and preventing such injuries.

Protein 144

Protein Medicine Research Genetics 144

pharmaphorum

FEBRUARY 16, 2021

Medical charity Alzheimer’s Research UK has teamed up with artificial intelligence specialist Exscientia to find new drug treatments for the devastating neurodegenerative disease. . It reckons its use of AI and machine learning can trim years off the current 12- to 15-year cycle from early research to marketed product.

Research 117

Research Drugs Protein Development 117

Scienmag

MAY 18, 2021

(Boston)–Researchers from Boston University School of Medicine (BUSM) report the formation of human cells containing a green fluorescent protein or GFP (one of the most important proteins in biology and fluorescence imaging) genetically fused with two interferon stimulated genes (ISGs), namely Viperin and ISG15.

Immune Response 41

Immune Response Reagent Research Protein 41

pharmaphorum

JANUARY 26, 2023

Both long- and short-read sequencing today is faster, more affordable, and highly accurate, providing researchers with deep insights to fuel research. Genomic insights will increasingly be used to reduce the hundreds of millions of pounds wasted administering drugs that are ineffective due to an individual’s genetic makeup.

Genome 128

Genome Genomics Genome Project Genetics 128

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.

Drugs 52

Drugs Gene Editing Genome Genomics 52

pharmaphorum

SEPTEMBER 16, 2022

Alphabet subsidiary and precision health company Verily recently announced a breakthrough in its AI drug discovery GPCR research collaboration with Sosei Heptares. Kim recognised then, he said, that better tools for medicine needed to be developed, and that they still do. What, then, is the solution?

Development 134

Development Research Drugs Clinical Trials 134

Drug Discovery World

NOVEMBER 1, 2022

Researchers say they have successfully used a cell’s natural process for making proteins to ‘slide’ genetic instructions into a cell and produce critical proteins missing from those cells. . Credit: Alexei Bygrave, Johns Hopkins Medicine. Splicing-linked expression design. in their effectiveness. “We

Gene Expression 52

Gene Expression Gene Packaging Packaging 52

Scienmag

NOVEMBER 18, 2020

— Research on a genetic heart disease has uncovered a new and unexpected mechanism for heart failure. This landmark discovery found a correlation between the clumping of RNA-binding proteins long linked to neurodegenerative disease ? ROCHESTER, Minn.

Genetics 40

Genetics RNA Protein Research 40

Advarra

JANUARY 11, 2024

The applications of mRNA-based therapies in cancer research represent one of the next groundbreaking steps toward improved cancer treatments. While mRNA usage has played several roles in clinical research , oncology researchers in particular are eager to explore the possibilities of mRNA-based cancer vaccines.

Vaccination 52

Vaccination Vaccine Protein Antibody 52

Scienmag

MAY 9, 2021

80% of all SARS-CoV-2 proteins produced in the laboratory — protocols available for worldwide research – Goethe University Frankfurt, Germany, forms the hub of research network from 17 countries Credit: Uwe Dettmar for Goethe-University Frankfurt, Germany FRANKFURT.

Research 57

Research Development Genetics Protein 57

Scienmag

MARCH 23, 2021

Additionally, […].

Genetics 41

Genetics Research Protein Medicine 41

Pharmaceutical Technology

APRIL 20, 2023

In addition to new regulatory functionalities, this feature may result in stronger and more durable protein expression. Circio aims to develop new circRNA medicines initially for cancer, then plans to expand rapidly into vaccines and gene therapy.

Gene Therapy 240

Gene Therapy RNA Gene Engineer 240

Drug Discovery World

DECEMBER 19, 2023

MPS I is a rare, genetic disease that affects the body’s ability to produce the enzyme alpha-L-iduronidase (IDUA). Developer Immusoft has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for the therapy, designated ISP-001, in this indication.

Engineer 113

Engineer Trials Genetic Disease Gene Therapy 113

Pharmaceutical Technology

MAY 25, 2023

The technology platforms include the Life Edit gene editing platform, an RNA, cell, protein, vector engineering and induced pluripotent stem cells (iPSCs) platform. ElevateBio intends to use the funds to advance its genetic medicine current good manufacturing practice (cGMP) and process development business, BaseCamp.

Gene Therapy 243

Gene Therapy Gene Gene Editing Manufacturing 243

Scienmag

JUNE 22, 2021

New research published in the Journal of Cellular and Molecular Medicine indicates that SARS-CoV-2, the virus that causes COVID-19, produces microRNAs that can have impacts on infected cells. Investigators found that the […].

Protein 42

Protein Genetics Medicine Production 42