Scienmag

NOVEMBER 22, 2021

Scientist are now combining recent advances in evolutionary analysis and deep learning to build three-dimensional models of how most proteins in eukaryotes interact.

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Protein Scientist Genetics Medicine 90

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

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.

Genetics 109

Genetics Drugs Protein Scientist 109

Pharmaceutical Technology

AUGUST 5, 2022

As per the prevailing prion theory, a misfolded version of a normal cell-surface protein acts as the chief infectious agent. It is ultra-rare,” says Jesús Rodriguez Requena, PhD, associate professor in the Department of Medicine at the University of Santiago de Compostela. And then comes the question of the right target.

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Drugs Protein Vaccine Vaccination 299

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

Pharma Mirror

APRIL 21, 2024

With the rapid development of biotechnology and molecular medicine, the introduction of mRNA as a vaccine or therapeutic agent enables the production of almost any desired functional protein/peptide within the human body.

In-Vitro 130

In-Vitro Drugs Genetic Disease RNA 130

Worldwide Clinical Trials

AUGUST 14, 2024

They play a crucial role in tailoring hematology oncology therapeutic strategies to individual patients and have transformed personalized medicine in oncology. This resistance may occur due to genetic mutations, epigenetic changes, or a tumor’s microenvironment.

Genetics 195

Genetics Protein Gene Genotype 195

Medical Xpress

DECEMBER 5, 2022

Biomedical and genetic engineers at Duke University and the Albert Einstein College of Medicine have designed a small fluorescent protein that emits and absorbs light that penetrates deep into biological tissue.

Protein 75

Protein Genetic Engineering Research Engineer 75

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. Here he gives us a deeper look at how genomic medicine is evolving and the barriers that are preventing it from reaching its full potential.

Genome 119

Genome Genomics Medicine Gene 119

Pharmaceutical Technology

JUNE 29, 2022

The next wave of medicine is well on course to be cell and gene-based. In September 2021, GlobalData figures revealed there to be 1,320 industry-sponsored regenerative medicine and advanced therapy trials ongoing worldwide. Meanwhile, the pharma industry is undergoing somewhat of a transformation itself.

Drugs 295

Drugs Gene Therapy Production Manufacturing 295

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

OCTOBER 1, 2020

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

Genetics 52

Genetics Medicine Research Protein 52

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.

Gene Editing 75

Gene Editing Genetics Hormones Medicine 75

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

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 Development 240

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 Development 243

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 145

Protein Medicine Research Genetics 145

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

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 […].

Genetics 73

Genetics Protein Medicine 73

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

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

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 75

DNA Protein Genetics Medicine 75

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

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.

Genome 129

Genome Genomics Genome Project Genetics 129

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.

Gene Editing 52

Gene Editing Genetics Drugs Gene 52

The Pharma Data

JULY 15, 2021

The access to the world’s largest browsable resource linking rare protein-coding genetic variants to human health and disease was launched through a genetic exome sequence analysis collaboration between AbbVie (NYSE: ABBV), Biogen Inc. Nasdaq: BIIB) and Pfizer (NYSE: PFE). The browser can be accessed via [link].

Biobanking 52

Biobanking Genetics Protein Medicine 52

The Pharma Data

JULY 8, 2021

Today, access to the world’s largest browsable resource linking rare protein-coding genetic variants to human health and disease was launched through a genetic exome sequence analysis collaboration between AbbVie (NYSE: ABBV), Biogen Inc. Nasdaq: BIIB) and Pfizer (NYSE: PFE). The browser can be accessed via [link]. About AbbVie.

Genetics 52

Genetics Biobanking Protein Containment 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 […].

Scientist 83

Scientist Gene RNA Protein 83

XTalks

FEBRUARY 7, 2025

The clinical-stage biopharmaceutical company focuses on developing precision medicines for chronic kidney disease (CKD), cardiovascular and metabolic conditions. Utilizing its proprietary Compass platform, Maze analyzes genetic data to create targeted therapies that address the root causes of disease.

Marketing 66

Marketing Genetics Vaccination Vaccine 66

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

Pharmaceutical Technology

AUGUST 17, 2022

oRNA molecules have been demonstrated to possess increased stability in vivo compared to linear mRNA and can potentially create more quantities of therapeutic proteins within the body. . By self-circularisation, Orna’s oRNA technology makes circular ribonucleic acids (oRNAs) from linear RNAs.

RNA 147

RNA Vaccination Vaccine In-Vivo 147

Pharmaceutical Technology

MAY 8, 2023

These viruses carry the genetic information to synthesise an RNA-dependent RNA polymerase. SsRNA virus peptide-derived molecules are attractive entities because they can mimic the structural features found in RNA-binding proteins. Peptide-based therapies are versatile, highly efficacious, and have a tolerable side effect profile.

RNA 240

RNA Genetics Vaccine Vaccination 240

pharmaphorum

JANUARY 21, 2021

This is the first project to be funded by the international Psychiatry Consortium, a £4 million collaboration between seven global pharmaceutical companies, and two leading research charities, convened and managed by the Medicines Discovery Catapult, that supports high-value drug discovery projects in this area of unmet patient need.

Medicine 111

Medicine Protein Drugs Research 111

Scienmag

DECEMBER 9, 2020

A single protein that appears necessary for the COVID-19 virus to reproduce and spread to other cells is a potential weakness that could be targeted by future therapies.

Protein 75

Protein Genetics Allergies Vaccination 75

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

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

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