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 227

Therapies Clinical Trials Trials Disease 227

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 291

Engineering Therapies Disease Research 291

Science Daily: Pharmacology News

APRIL 9, 2025

By inserting an entire gene, the tool offers a one-and-done approach that overcomes hurdles from CRISPR gene editing technology -- which is programmed to correct individual mutations -- offering a promising step forward for gene therapy.

RNA 252

RNA DNA Disease Therapies 252

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 231

Therapies Clinical Trials Treatment Disease 231

Science Daily: Pharmacology News

APRIL 9, 2025

Researchers have discovered a way to get anti-inflammatory medicine across the blood-brain barrier, opening the door to potential new therapies for a range of conditions, including Alzheimer's disease, multiple sclerosis, Parkinson's disease and cancer cachexia.

Treatment 279

Treatment Therapies Disease Research 279

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 144

Science RNA Therapies Disease 144

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 342

Therapies Disease Research 342

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 211

Disease Drug Development Therapies Drugs 211

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 348

Therapies Disease Research Drugs 348

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.”

Clinical Trials 128

Clinical Trials Trials Pharmaceuticals Drug Development 128

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.

Therapies 309

Therapies Disease Research 309

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

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 310

Disease Therapies Treatment 310

Drug Target Review

APRIL 7, 2025

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Safety biomarkers account for adverse effects of a therapy under study. There are several types of biomarkers to consider.

Clinical Development 69

Clinical Development Clinical Trials Clinical Pharmacology Trials 69

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

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

Science Daily: Pharmacology News

JANUARY 26, 2024

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

Therapies 243

Therapies Disease Research 243

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

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?

Trials 87

Trials Disease Clinical Trials Therapies 87

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.

Therapies 118

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

Disease 124

Disease Research Science Medical Schools 124

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 105

Therapies FDA Clinical Trials Disease 105

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

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 Research 105

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 […]

Research 82

Research Therapies Disease Doctors 82

Drug Target Review

FEBRUARY 1, 2024

What challenges has the industry faced in developing and commercialising treatments for neurological diseases, including rare diseases? There are a lot of drug development challenges that are, in some cases, unique to neuroscience, including neurological diseases, psychiatric disorders, and rare diseases affecting the nervous system.

Therapies 104

Therapies Drug Development Disease Clinical Development 104

DrugBank

JANUARY 9, 2025

Targeted Therapies vs. Traditional Treatments Traditional cancer treatments, such as chemotherapy and radiotherapy, are inherently cytotoxic, targeting both cancerous and healthy cells. This lack of selectivity often results in significant side effects, including nausea and limited efficacy, particularly in advanced or metastatic disease.

Therapies 52

Therapies Disease Treatment Small Molecule 52

PPD

JANUARY 15, 2025

As clinical trials become increasingly complex, particularly in decentralized trials and rare disease studies, sponsors experience increased challenges in site selection, forecasting and resourcing, and patient recruitment and enrollment.

Clinical Trials 95

Clinical Trials Trials Clinical Research Drug Development 95

Drug Target Review

APRIL 14, 2025

A deep interest in political science and the political economy of drugs led him to focus on how drugs are priced and distributed across different jurisdictions. As CEO, he oversees everything except the science, which is driven by his co-founder, Jim Roberts. Finrows role at Lumen Bioscience reflects his diverse expertise.

Therapies 52

Therapies Marketing Drug Development Treatment 52

Drug Target Review

FEBRUARY 28, 2024

Over the past 25 years, T-cell therapies have gained significant ground in the treatment of cancer. Preclinical research on γδ T cells has made great strides since the cells were first identified in the 1980s, with γδ T-cell therapies from several companies, including IN8bio, now in or nearing clinical trials for various cancers.

Therapies 105

Therapies Engineering Molecular Biology Science 105

SCIENMAG: Medicine & Health

OCTOBER 2, 2023

Researchers at the University of Cologne’s CECAD Cluster of Excellence for Aging Research and the CEPLAS Cluster of Excellence for Plant Sciences have found a promising synthetic plant biology approach for the development of a therapy to treat human neurodegenerative diseases, especially Huntington’s disease.

Therapies 52

Therapies Disease Science Research 52