Worldwide Clinical Trials

JANUARY 6, 2025

Tom Zhang, PhD, Chief Scientific Officer, Large Molecule Bioanalysis Revolutionizing Therapeutic Development with Cutting-Edge Bioanalysis Automation Cutting-edge advancements in bioanalysis (BioA) and drug R&D are reshaping the future of preventative and interventional care, but how do we harness these to their fullest potential?

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Clinical Trials Clinical Trials Trials Genetics 130

Pharmaceutical Technology

MARCH 2, 2023

The US Food and Drug Administration (FDA) has approved Reata Pharmaceuticals ’ oral, once-daily medication SKYCLARYS (omaveloxolone) to treat Friedreich’s ataxia patients. SKYCLARYS is the first approved treatment in Friedrich’s Ataxia and marks a milestone for drug development in this complex disease.

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FDA Approval Trials Genetics Drugs 264

Worldwide Clinical Trials

OCTOBER 14, 2024

Scientists now use cutting-edge techniques, including biological therapies that target specific immune system components and precision medicine approaches that personalize treatment plans based on a patient’s genetic profile.

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Research Clinical Trials Clinical Trials Immune Response 147

Worldwide Clinical Trials

OCTOBER 14, 2024

Scientists now use cutting-edge techniques, including biological therapies that target specific immune system components and precision medicine approaches that personalize treatment plans based on a patient’s genetic profile.

Research 130

Research Clinical Trials Clinical Trials Immune Response 130

XTalks

MAY 20, 2022

If you enjoy working with biological samples and are enthusiastic about healthcare, a career as a clinical scientist might be just right for you. To start applying to clinical scientist jobs today, head over to the Xtalks Job Search platform. Who is a Clinical Scientist? What is it Like to Work as a Clinical Scientist?

Scientist 105

Scientist Life Science Clinical Research Doctors 105

pharmaphorum

MARCH 21, 2024

Surgeons in the US have carried out the world’s first transplant of a pig kidney into a human, a feat made possible by genetic modification of the organ and an experimental immunosuppressant drug regimen to prevent rejection.

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Scientist Genetics Drugs 110

pharmaphorum

NOVEMBER 12, 2021

Researchers in the US have developed an artificial intelligence-based tool that is able to predict COVID-19 symptoms and suggest which FDA-approved drugs might be used to treat patients. The tool meanwhile could also be applied to new outbreaks of other novel viral infections as they emerge, according to the scientists.

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Doctor Doctors Drugs Gene Expression 135

Medical Xpress

FEBRUARY 13, 2023

The behavioral disorders observed in autism are associated with a multitude of genetic alterations. Scientists from the Hector Institute for Translational Brain Research (HITBR) have now found another molecular cause for this condition.

Genetics 98

Genetics Scientist Drugs Research 98

Pharmaceutical Technology

JULY 7, 2022

Genetic mutations, both germline and acquired, are behind a large proportion of the most debilitating and sometimes life-threatening human diseases. But scientists have struggled to find effective treatments for many of these diseases since the dawn of modern medicine. Securing the supply chain.

Gene Therapy 245

Gene Therapy Gene Medicine Genetics 245

pharmaphorum

OCTOBER 12, 2021

A data-mining study conducted by researchers in the US has found that an already-approved diuretic drug could have potential as a treatment for some patients with Alzheimer’s disease. The results are strong enough to back a proof-of-concept study in people with genetic risk of Alzheimer’s according to the researchers.

Gene Expression 111

Gene Expression Drugs FDA Approval Gene 111

Scienmag

DECEMBER 30, 2021

Immunotherapies called chimeric antigen receptor (CAR) T cells use genetically engineered versions of a patient’s own immune cells to fight cancer. Now, scientists at Memorial Sloan Kettering Cancer Center’s Sloan Kettering Institute (SKI) have developed new CAR T cells that can do something their […].

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Scientist Genetic Engineering Engineer Genetics 61

Scienmag

DECEMBER 9, 2020

Credit: Phil Robinson Scientists have produced a tomato enriched in the Parkinson’s disease drug L-DOPA in what could become a new, affordable source of one of the world’s essential medicines. This novel use of tomato […].

Drugs 75

Drugs Genetics Scientist Medicine 75

STAT News

JANUARY 12, 2023

Nearly 25 years later, Sonia Vallabh and her husband, Eric Minikel, quit their jobs — she as a lawyer, he as a programmer and analyst — and retrained as scientists to find cures for the genetic prion disease that killed Vallabh’s mother and that she herself is at high risk of developing.

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Development Drugs Genetics Scientist 98

Medical Xpress

MAY 1, 2023

A decade after scientists developed the ability to edit DNA using the CRISPR sequence, the first drugs using the technique are approaching the market, with the potential to transform the lives of people with certain genetic illnesses. But questions of ethics, access, and pricing remain.

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DNA Genetics Scientist Marketing 90

pharmaphorum

OCTOBER 2, 2020

Swiss medical data specialist Sophia Genetics has raised $110 million in an oversubscribed funding round that will be used to boost its headcount and international presence and prepare to take its shares public. The post $110m financing sets up US, Asia expansion for Sophia Genetics appeared first on.

Genetics 59

Genetics Genomics Genome Life Science 59

Scienmag

MARCH 2, 2021

Process paves a road to safe, ethical, and fast drug manufacturing Credit: Rensselaer Polytechnic Institute TROY, N.Y. — Envisioning an animal-free drug supply, scientists have — for the first time — reprogrammed a common bacterium to make a designer polysaccharide molecule used in pharmaceuticals and nutraceuticals.

Bacteria 86

Bacteria Bacterium Drugs Scientist 86

pharmaphorum

DECEMBER 24, 2020

A serotonin sensor designed using Artificial Intelligence (AI) could help scientists study sleep and mental health and potentially find new neurology drugs. The technique could also be used to test the effectiveness of new psychoactive drugs, according to the US-government funded NIH.

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Development Drugs Protein Genetic Engineering 77

XTalks

OCTOBER 19, 2023

An assay development scientist combines deep biological and biochemical expertise with practical laboratory skills to develop tests that can answer specific biological questions or drive the drug discovery process. Related: Pharmaceutical Scientist Jobs: What to Know Before Applying What Does an Assay Development Scientist Do?

Scientist 59

Scientist Development Research Compliance 59

Pharmaceutical Technology

APRIL 26, 2023

The US Food and Drug Administration (FDA) has granted accelerated approval for Biogen ’s Qalsody (tofersen) 100mg/15mL injection to treat adult amyotrophic lateral sclerosis (ALS) patients with a mutation in the superoxide dismutase 1 (SOD1) gene. It is reported to be the first treatment targeting a genetic cause of ALS to receive approval.

Genetics 130

Genetics Protein Trials Gene 130

Medical Xpress

JANUARY 5, 2023

Scientists at St. Jude Children's Research Hospital are reporting the most comprehensive study to date describing the variations in drug response across different genetic subtypes of acute lymphoblastic leukemia (ALL). The findings provide a blueprint for precision medicine to further individualize therapy.

Medicine 75

Medicine Genetics Scientist Drugs 75

Medical Xpress

FEBRUARY 23, 2023

Scientists at Open Targets, EMBL's European Bioinformatics Institute (EMBL-EBI), and GSK are revealing the shared basis of diseases using a map of interacting human proteins.

Bioinformatics 75

Bioinformatics Drugs Protein Scientist 75

Scienmag

NOVEMBER 20, 2020

Scientists at Goethe University within the international consortium COVID19-NMR refine previous 2D models The genetic code of the SARS-CoV2 virus is exactly 29,902 characters long, strung through a long RNA molecule. It contains the information for the production of 27 proteins.

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Genome Genomics RNA Protein 70

NY Times

JUNE 28, 2021

A new workshop explores the right of Indigenous people to govern the collection, ownership and use of their biological and cultural data.

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Scientist Genetics Drugs Marketing 93

Pharma Focus Asia

SEPTEMBER 12, 2022

With the recent advancements in technology, scientists are able to gather and analyze more data than ever before. This has allowed researchers to create drugs that target specific populations and individual patients using biotechnology and genetic data. Here are some of the ways that technology is helping […].

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Drugs Genetics Scientist Research 52

Pharmaceutical Technology

SEPTEMBER 29, 2022

The alliance will leverage the CRISPR genome editing technologies of Scribe to facilitate in genetic modification of new natural killer (NK) cell therapeutics for cancer. According to the deal, Sanofi will make an upfront payment of $25m to Scribe. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Development In-Vivo Genome Genomics 130

Scienmag

SEPTEMBER 22, 2020

Patients with BRCA1/2 mutations are at higher risk for breast, ovarian and prostate cancers that can be aggressive when they develop – and, in many cases, resistant to lifesaving drugs.

Drugs 69

Drugs Scientist Development Genetics 69

Pharmaceutical Technology

JULY 5, 2022

In addition, the scientists later found that the HAV needs TENT4A/B for its replication. Subsequently, Lemon’s lab tested RG7834 and found the precise effects of the oral drug on HAV in liver and faeces. The latest findings are the first to show that a drug was effective against HAV in an animal model of the disease.

Protein 130

Protein RNA Genomics Genome 130

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?driven,

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Gene Editing Bacteria DNA Gene 105

Pharmaceutical Technology

NOVEMBER 29, 2022

These viruses are engineered genetically for killing cancer. Using chemokines, the scientists made the GBM tumours “hot”, making the chemokines aid in the immune cell migration to the tumour microenvironment. “So, GBMs are called “cold” tumours as they lack helpful immune cells.

Development 130

Development Research Antibody Engineer 130

Bioengineer

JULY 22, 2021

Geneticists from Trinity College Dublin have discovered how a specific genetic mutation called H3K27M causes a devastating, incurable childhood cancer, known as diffuse midline glioma (DMG), and – in lab studies working with model cell types – successfully reverse its effects to slow cancer cell growth with a targeted drug.

Genetics 52

Genetics Drugs Clinical Trials Clinical Trials 52

XTalks

FEBRUARY 2, 2022

The Israeli Society of Gene and Cell Therapy (ISGCT) is hosting its upcoming annual meeting in-person this year to encourage networking and the sharing of information about genetic and cellular therapies. As of now, the US Food and Drug Administration (FDA) has approved 22 gene and cell therapy products.

Gene 98

Gene Research Genetics Clinical Trials 98

Cloudbyz

MARCH 5, 2024

In the intricate dance of drug discovery and development, two protagonists emerge as the cornerstones of modern medicine: biologics and small molecules. Manufacturing Process : Small Molecules: Manufactured through chemical synthesis, small molecule drugs can be consistently reproduced with a high degree of purity and stability.

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Development Drugs Immune Response Manufacturing 97

The Pharma Data

NOVEMBER 17, 2021

Mount Sinai researchers have developed a new model that uses DNA and RNA sequencing data from hundreds of patients to identify specific genes and genetic alterations responsible for never-before-defined subtypes of a blood cancer called multiple myeloma. doi: 10.1126/sciadv.abg9551. Source link: [link].

Scientist 52

Scientist RNA DNA Genetics 52

Scienmag

DECEMBER 9, 2020

A new genetic approach can accelerate the study of phage-microbe interactions with implications for health, agriculture, and climate Credit: Wikimedia Commons Scientists are continually searching for new and improved ways to deal with bacteria, be it to eliminate disease-causing strains or to modify potentially beneficial strains.

Bacteria 52

Bacteria Genetic Engineering Genetics Engineer 52

pharmaphorum

OCTOBER 7, 2022

Two additional novel AI-generated targets for chronic kidney disease (CKD) and idiopathic pulmonary fibrosis (IPF) have been discovered using the Benevolent Platform and selected for AstraZeneca’s (AZ) drug discovery portfolio. BenevolentAI is a leading clinical-stage AI drug discovery company.

Drugs 104

Drugs Clinical Trials Clinical Trials Genetics 104

STAT News

NOVEMBER 14, 2022

In this video, we chat with health policy and drug pricing researcher Hussain Lalani; Julia Joung, a molecular biologist developing genetic screening technologies; and Avinash Manjula Basavanna, who researches engineered living materials and has developed a printable ink made from bacteria.

Bacteria 96

Bacteria Engineer Genetics Research 96

XTalks

FEBRUARY 4, 2021

The power of leveraging clinical data to decipher disease mechanisms and fuel drug discovery has rapidly grown in the era of genomics and personalized medicine. Biobanking will continue to play an integral role in accelerating drug discovery and development, which will necessitate access to a larger array of biological samples.

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Research Biobanking Drugs Genomics 98