Science Daily: Pharmacology News

JULY 12, 2023

They also found that co-transplantation of neuronal cell therapy with host regulatory T cells resulted in effective suppression of needle trauma and significant improvement in the survival and recovery of grafts. These findings suggest a path for the 'realistic' use of cell therapy to treat neurodegenerative disorders.

Therapies 189

Therapies Immune Response Disease Research 189

Science Daily: Pharmacology News

NOVEMBER 13, 2023

This discovery opens up new opportunities for therapeutic intervention to control cholesterol uptake that could complement other therapies and potentially save lives. Researchers have discovered the mechanism by which cholesterol in our diet is absorbed into our cells.

Therapies 236

Therapies Disease Research 236

Science Daily: Pharmacology News

AUGUST 30, 2023

A clinical trial has tested a potentially curative stem cell gene therapy for sickle cell disease. The results were promising.

Therapies 217

Therapies Clinical Trials Trials Disease 217

Science Daily: Pharmacology News

MARCH 13, 2025

Such neurons could be used to treat spinal cord injuries or diseases such as ALS. Researchers devised a process to convert a skin cell directly into a neuron, eliminating the need to generate induced pluripotent stem cells.

Engineering 262

Engineering Therapies Disease Research 262

Science Daily: Pharmacology News

FEBRUARY 22, 2024

A large-scale clinical trial of treatment strategies for Crohn's disease has shown that offering early advanced therapy to all patients straight after diagnosis can drastically improve outcomes, including by reducing the number of people requiring urgent abdominal surgery for treatment of their disease by ten-fold.

Therapies 224

Therapies Clinical Trials Treatment Disease 224

Drug Target Review

AUGUST 28, 2024

What are the main challenges currently faced in the treatment of chronic liver diseases, and how does Resolution Therapeutics aim to address these challenges? Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years.

Therapies 116

Therapies Disease Engineering Medical Schools 116

BioPharma Drive: Drug Pricing

JUNE 3, 2024

With new modalities such as gene and cell therapies, RNA therapeutics, complex biologics and more, today’s science brings unprecedented opportunities to address diseases that have long remained out of reach.

Science 143

Science RNA Therapies Disease 143

Science Daily: Pharmacology News

JANUARY 3, 2025

The research could revolutionize therapies for complex conditions like autoimmune disease and cancer. Bioengineers have developed a new construction kit for building custom sense-and-respond circuits in human cells.

Therapies 337

Therapies Disease Research 337

Science Daily: Pharmacology News

JULY 4, 2024

The new T cell atlas is publicly available and should help in the development of new drug therapies for immune-mediated diseases. The discoveries were made possible by a newly developed technology they call ReapTEC.

Therapies 345

Therapies Disease Research Drugs 345

Science Daily: Pharmacology News

NOVEMBER 28, 2023

Heart disease kills 18 million people each year, but the development of new therapies faces a bottleneck: no physiological model of the entire human heart exists -- so far.

Disease 204

Disease Drug Development Therapies Drugs 204

Drug Target Review

DECEMBER 20, 2024

Their work focuses on creating ‘digital twin generators’ – AI-driven models that predict how a patients disease may progress over time. As Smith puts it, “now the viewpoint is that computational science is very powerful, and these AI/ML methods are going to be transformative in a lot of ways.”

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Clinical Trials Trials Pharmaceuticals Drug Development 128

Drug Target Review

AUGUST 31, 2023

In the pursuit of a remedy for Alzheimer’s disease, a frontier in medical science is illuminating a glimmer of hope. Stem cell therapies have already demonstrated their prowess in treating diverse cancers and ailments linked to the blood and immune system.

Therapies 105

Therapies Disease Treatment Research 105

Broad Institute

MAY 16, 2024

Gene therapy could potentially treat a range of severe genetic brain disorders, which currently have no cures and few treatment options. Because the vehicle binds to a well-studied protein in the blood-brain barrier, the scientists say it has a good chance at working in patients.

Therapies 137

Therapies FDA Approval Engineering Disease 137

Science Daily: Pharmacology News

JUNE 10, 2024

The advance could one day help researchers develop a single gene therapy for diseases such as cystic fibrosis that are caused by one of hundreds or thousands of different mutations in a gene.

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Therapies Disease Research 298

Science Daily: Pharmacology News

FEBRUARY 14, 2024

A groundbreaking scientific study has unveiled a remarkable discovery that may have far-reaching implications for the treatment of heart disease. The implications are immense offering glimpses of a future where heart disease may no longer be an irreversible condition but a challenge that can be overcome through medical intervention.

Disease 300

Disease Therapies Treatment 300

Drug Target Review

MARCH 4, 2025

Phenomix Sciences , led by CEO Mark Bagnall, is using precision medicine to make treatments more targeted and effective. Phenomix Sciences, built on over a decade of clinical research at the Mayo Clinic, is disrupting this outdated approach. Obesity treatment is undergoing a major shift, much like the advances seen in cancer care.

Therapies 52

Therapies Treatment Clinical Trials Science 52

Drug Target Review

JANUARY 8, 2024

It is no mystery that as we age our health starts to deteriorate, and we become increasingly susceptible to diseases. Through years of scientific exploration and research, we now know there are several biological changes that make our bodies more susceptible to disease or injury, and we can target those with therapeutic interventions.

Science 145

Science Disease FDA Approval Clinical Trials 145

Codon

FEBRUARY 13, 2024

Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. alone, according to the Centers for Disease Control and Prevention.

Therapies 128

Therapies Virus DNA Clinical Trials 128

Drug Target Review

OCTOBER 16, 2023

How can PDOs revolutionise drug discovery and deepen our understanding of disease? Patient-derived organoids (PDOs) are proliferative 3D cell structures derived from tissue samples of both healthy and diseased tissue. Improved translatability to the patient and the disease state.

Disease 122

Disease Immune Response Cell Biology Therapies 122

Drug Target Review

MARCH 3, 2025

The search for effective treatments for neurodegenerative diseases like Parkinson’s disease has long been hindered by the brain’s complexity and the absence of adequate models for drug discovery. “For diseases like Parkinson’s, it’s more than sufficient,” he explains.

Disease 52

Disease Treatment Therapies Cell Biology 52

FDA Law Blog: Drug Discovery

JULY 17, 2024

Importantly, the Hub is intended to establish a new model within FDA, which leverages cross-Agency expertise in providing guidance and conducting reviews for products for rare disease populations. Early Direction for the Rare Disease Innovation Hub The Hub, which will be co-chaired by Drs. By Sarah Wicks & James E.

FDA 105

FDA Disease Clinical Pharmacology Science 105

Broad Institute

SEPTEMBER 6, 2023

Diabetes and cardiac fibrosis are two conditions in dire need of new therapies,” said Todd Golub, director of the Broad Institute. For both patient populations, there are no safe and effective therapies for reversing disease. The collaboration will focus on advancing three programs over the next three years.

Disease 136

Disease Research Therapies Treatment 136

Covalent Modifiers

SEPTEMBER 7, 2023

Science 2023 , 380, 1349-1356 DOI: 10.1126/science.adh0614 Millions who live in Latin America and sub-Saharan Africa are at risk of trypanosomatid infections, which cause Chagas disease and human African trypanosomiasis (HAT). Srinivasa P.

Treatment 173

Treatment Disease Science Therapies 173

Drug Target Review

NOVEMBER 8, 2023

Problem w/ CTs and foundational understanding of Vittoria: can you explore the current limitations of cell therapies and the challenges faced by patients and providers? Currently, only a small percentage of cancers can be effectively treated with cell therapies, and there is little diversity in the currently approved products.

Therapies 116

Therapies Engineering Treatment Molecular Biology 116

Science Daily: Pharmacology News

JANUARY 26, 2024

Researchers identified a retinal disease to evaluate the success of gene and cell replacement therapy.

Therapies 233

Therapies Disease Research 233

Fierce BioTech

SEPTEMBER 5, 2024

How can life sciences organizations effectively identify and recruit participants with specific genetic variants? How can life sciences organizations effectively identify and recruit participants with specific genetic variants? How can they engage providers and raise awareness of gene therapies for the patients who need them most?

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Trials Disease Clinical Trials Therapies 87

PLOS: DNA Science

OCTOBER 19, 2023

Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.

Therapies 107

Therapies FDA Clinical Trials Disease 107

Drug Target Review

NOVEMBER 17, 2023

They do not induce ‘graft versus host’ disease when transplanted for allogeneic therapy and there appears little sign of immune rejection. Peripheral blood NK cells have been used in allogeneic cancer therapies and shown to be safe. NK cells can be readily extracted from umbilical cord blood or peripheral blood of adult donors.

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Therapies Clinical Trials Cell Biology Trials 118

Broad Institute

JANUARY 25, 2024

They found significant associations between the thinning of different retinal layers and increased risk of developing ocular, cardiac, pulmonary, metabolic, and neuropsychiatric diseases and identified genes that are associated with retinal layer thickness. Their findings are published in Science Translational Medicine. “We

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Disease Research Science Medical Schools 124

PLOS: DNA Science

MARCH 21, 2024

The Food and Drug Administration just announced approval of Lenmeldy (atidarsagene autotemcel), a gene therapy to treat the neurological condition metachromatic leukodystrophy (MLD). The history of gene therapy for MLD is compelling – DNA Science covered it for Rare Disease Day in 2021, here.

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Therapies Clinical Trials DNA Trials 98

Covalent Modifiers

JANUARY 29, 2024

3c01835 Building on recent advances in peptide science, medicinal chemists have developed a hybrid class of bioconjugates, called peptide–drug conjugates, that demonstrate improved efficacy compared to peptides and small molecules independently. Moore Journal of Medicinal Chemistry 2024 DOI: 10.1021/acs.jmedchem.3c01835

Small Molecule 173

Small Molecule Drugs Science Disease 173

Drug Target Review

DECEMBER 13, 2024

These types differ in their etiology, natural history, and present distinct challenges in disease management. 2 Unmet needs in lung cancer treatment Recent decades have seen significant advancements in lung cancer treatment, especially with the introduction of targeted therapies and immunotherapies, which have notably improved survival rates.

Therapies 98

Therapies Treatment Clinical Trials Drugs 98

Drug Target Review

OCTOBER 30, 2024

Imagine being able to create an in vitro replica of a diseased organ to study the molecular mechanism underlying the illness. Now take a step further: envision testing drugs in these organoids to identify the ones that can treat disease safely and effectively without needing to run expensive clinical trials first.

Treatment 105

Treatment Disease Clinical Trials Molecular Biology 105

Drug Target Review

JUNE 21, 2023

Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.

Disease 111

Disease Clinical Trials Therapies Science 111

National Institute on Drug Abuse: Nora's Blog

FEBRUARY 1, 2024

50 years after founding, NIDA urges following science to move beyond stigma area Thu, 02/01/2024 - 11:20 Nora's Blog February 1, 2024 Image NIDA Image In 2024, NIDA celebrates its 50th anniversary. The knowledge generated by NIDA research has also led to a robust and rigorous science of drug use prevention.

Science 122

Science FDA Approval Treatment Research 122

Drug Target Review

JULY 7, 2023

Approximately three percent of the global population — 240 million people — experience autoantibody diseases, which occur when one’s own body attacks critical organs and tissues. This can create an abnormal immune response that attacks the cells of our bodies and contributes to the development of autoantibody diseases.

Disease 98

Disease Immune Response Therapies Treatment 98

SCIENMAG: Medicine & Health

JULY 28, 2023

Lynch, lead author, is now a research fellow in the Department of Veterinary and Animal Sciences. Pearson, co-author of the paper, is now a NEWVEC post-doctoral researcher at UMass Amherst. Credit: UMass Amherst […]

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Research Therapies Disease Doctors 82