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.

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FDA Approval Genetics Gene Therapy Gene 105

Pharmaceutical Technology

MAY 17, 2023

It will use the precision medicine approach, as well as the deep expertise of Chinook Therapeutics in nephrology and knowledge of Ionis Pharmaceuticals in RNA-targeted therapeutics. Antisense therapies are designed for destroying mRNA, reducing the amount of disease-causing protein.

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Development Protein RNA Genetics 246

Scienmag

OCTOBER 1, 2020

Credit: Edward Lee PHILADELPHIA — A new, rare genetic form of dementia has been discovered by a team of Penn Medicine researchers.

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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

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

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.

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Protein Manufacturing Gene Sequencing Production 52

Scienmag

APRIL 15, 2021

Team of Japanese and European scientists identify a novel genetic mitochondrial disorder by analyzing DNA samples from three distinct families Credit: Fujita Health University DNA ligase proteins, which facilitate the formation of bonds between separate strands of DNA, play critical roles in the replication and maintenance of DNA.

Genetics 105

Genetics DNA Genome Genomics 105

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.

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Protein Gene Life Science Research 98

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.

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Gene Editing Genetics Hormones Medicine 75

XTalks

FEBRUARY 11, 2021

This has ushered in a new era of genomics that is fostering rapid, detailed and personalized insights into human genetics. Xtalks is celebrating the International Day of Women and Girls in Science with a special overview of notable female scientists who have made revolutionary contributions to the field of genetics. Rosalind Franklin.

Genetics 119

Genetics DNA Genome Genomics 119

pharmaphorum

MARCH 26, 2021

There’s a new kid on the block among companies using artificial intelligence in drug discovery, after 1910 Genetics launched today with $26 million in financing. 1910 Genetics is named after the year in which sickle cell disease (SCD) was first discovered in the US by James Herrick. Dr Jen Nwankwo.

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Genetics Drugs Protein Scientist 87

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

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.

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Protein Genetics Medicine Research 40

Pharmaceutical Technology

JULY 6, 2022

A protein-based vaccine, NVX-CoV2373 is made from the genetic sequence of the SARS-CoV-2 virus’ first strain. The latest development comes after the Committee for Medicinal Products for Human Use of the European Medicines Agency granted a positive recommendation in June this year.

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Vaccination Vaccine Protein Clinical Trials 264

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. The presence of these biomarkers could be used to accelerate the development of more personalised treatments for triple negative breast cancers.

Genetics 52

Genetics Gene Hormones RNA 52

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.

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Gene Editing Genetics Drugs Gene 52

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.

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DNA Protein Genetics Medicine 74

BioPharma Reporter

OCTOBER 17, 2023

SpliceBio, a genetic medicines company harnessing protein splicing to develop the next generation of gene therapies, has partnered with Spark Therapeutics to develop a gene therapy for an inherited retinal disease.

Gene 59

Gene Gene Therapy Development Genetics 59

Scienmag

MARCH 9, 2022

The genome also has the code for making a capsid, the protein shell of a virus that encapsulates the genome and protects it like a nanocontainer. . — Each simple RNA virus has a genome, its “native RNA.” This genome dictates how the virus replicates in cells to eventually cause disease. Credit: Zandi lab, […].

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Packaging Packaging Genetics Genome 84

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.

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Genetics Production Development Drugs 59

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

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.

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Development Genetic Disease Genetics Gene Therapy 130

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.

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Protein Genetic Disease Genetics Scientist 42

Scienmag

NOVEMBER 25, 2020

An international study, coordinated by experts from the University of Nottingham, has revealed that the genetic risk of pre-eclampsia – a potentially dangerous condition in pregnancy – is related to blood pressure and body mass index. It […].

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Genetics Protein Medicine 73

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.

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Genetics RNA Protein Research 40

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 (..)

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RNA Protein Drugs Research 87

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

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

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.

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Protein RNA Genome Genomics 130

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

Scienmag

APRIL 27, 2022

CHAPEL HILL, NC – Inside embryonic cells, specific proteins control the rate at which genetic information is transcribed from DNA to messenger RNA – a crucial regulatory step before proteins are created. Those specific “regulatory” proteins are called transcription factors, and they do their thing by binding to […].

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Scientist Gene RNA Protein 83

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

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.

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Protein Medicine Research Genetics 144

Drug Discovery World

JANUARY 9, 2024

On Monday 5 February at 3.30pm, he will speak on ‘ Proximity-inducing drugs: using technological innovation to build the next generation of medicine. Simon Bushell DDW is delighted to announce that the programme will feature Simon Bushell, SVP of Molecular Discovery of Entact Bio.

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Drugs Protein Clinical Trials Clinical Trials 64

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.

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Research Drugs Gene Therapy Genetics 52

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 Regulation Gene 52

pharmaphorum

JANUARY 26, 2023

We are already seeing an increase in projects exploring population genomics in Africa, the Middle East, and Asia, with initiatives including the GenomeAsia100K Project and the Genome Aggregation Database focusing on capturing genetic data of non-European individuals. Just one mutation can lead to a complete change in protein formation.

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