Research on discovering and developing new drugs. Covers structure-revealing tools, eye-disease mechanisms, protein screening, and once-daily oral medicines.
The ISSCR Consortium supports the FDA's draft guidance on using new approach methodologies (NAMs) in drug development, emphasizing the need for a flexible and science-driven framework. The consortium recommends clarifying biological complexity alignment with context of use and expanding recognition of computational modeling and hybrid ...
Researchers used wastewater surveillance to detect drug-resistant Candida auris strains up to five months before patients showed symptoms, opening a new frontier for hospitals. The study found higher detection rates and concentrations of the pathogen in hospital wastewater compared to community-scale treatment plants.
Researchers developed a new method called sm-PORE-cupine to study individual RNA molecules and reveal how their structures influence gene regulation. The technology provides deeper insight into how RNA structure affects cellular function and could help identify new therapeutic targets for diseases such as viral infections.
Researchers developed ApexGO, an AI-powered method to turn weak antibiotic candidates into more potent ones. The tool uses generative AI and Bayesian optimization to guide molecular tweaks, predicting which changes are likely to increase antimicrobial activity.
A PSU-led research team has developed a novel chemical compound, T111, that shows promise for treating and preventing malaria. The compound targets all three major life-cycle stages of the malaria parasite, making it a strong candidate for a single-encounter radical cure.
Researchers developed genESOM, a generative AI that can expand dataset volume and simulate larger animal numbers while maintaining reliability. This allows for 30-50% reduction in animal experiments without compromising results.
A team of researchers has uncovered a promising new target for antimalarial drug design, identifying an enzyme called aminopeptidase P from the Plasmodium falciparum parasite. The new inhibitors have been shown to bind more strongly and selectively than existing compounds, demonstrating potential as a new class of drugs to combat malaria.
The UK-led OpenBind initiative has released its first publicly available dataset and predictive AI model, accelerating the discovery of new medicines using artificial intelligence. The release showcases high-quality, standardized experimental data and a trained predictive model, enabling researchers worldwide to drive the next generati...
A multi-institutional team led by Sanford Burnham Prebys aims to develop a non-opioid pain therapeutic using lead molecule SBI-810. The effort, funded by a $3.9 million NIH grant, seeks to optimize the compound into a drug that could provide effective pain relief without addiction risks.
Researchers have developed a novel method to generate diazo compounds without toxic precursors, enabling efficient synthesis of valuable intermediates for chemical and pharmaceutical applications. The phosphine-mediated Michael addition reaction produces β-heteroatom-substituted diazo esters under mild conditions.
The FDA has merged two of OYE Therapeutics' Investigational New Drug applications, creating a more streamlined regulatory framework for development across related programs. This move is expected to improve regulatory efficiency, reduce duplicative development activity, and simplify operational execution.
A research consortium has developed mini-antibodies that can reactivate mutated p53, a key tumor suppressor protein. These DARPins bind selectively to p53 mutants, restoring stability and functionality, making them potentially useful against various types of tumors.
Volume 39 of SLAS Discovery highlights novel assays and AI-assisted workflows to accelerate personalized cancer immunotherapy. The journal aims to advance life sciences discovery through education, knowledge exchange, and global community building.
Chronic liver disease in the US costs $41.57 billion, with prescription medication expenditures accounting for nearly half of all-cause healthcare expenditures. Poor adherence to antiviral medications is a key factor in preventing disease progression and treatment, with high out-of-pocket costs increasing non-adherence risk.
Researchers at the University of East Anglia have discovered that light can be programmed using its natural geometry, allowing for the creation of structured light with unique properties. This breakthrough has far-reaching implications for fields such as medicine, data transmission, and quantum technologies.
The new facility enables scientists to observe and measure detonation forces in unprecedented detail, shedding light on industrial safety risks and potential breakthroughs. Researchers aim to develop safer designs and protocols by examining detonation disasters like the Buncefield Fire.
Dr. Dennis Slamon, a renowned breast cancer researcher, has been elected to the Association of American Physicians (AAP) for his pioneering work in HER2 gene discovery and targeted therapies. His research led to significant improvements in survival rates and established the principle of targeted cancer treatment.
A computational protocol has been established by University of Kent researchers to accurately identify reactions that can result in successful drug candidates for Chagas disease. This approach reduces the need for trial-and-error, prioritizing promising compounds earlier and making the drug discovery process faster and more affordable.
The experimental drug DH20931 targets triple-negative breast cancer by triggering a surge in fat-like molecules called ceramides, overwhelming cancer cells with toxic fats. In lab experiments, the drug made standard chemotherapy more effective, reducing the dose needed to kill cancer cells by about fivefold.
Researchers have discovered a way to prevent the collaboration between two important cancer-related proteins, N-MYC and Aurora A. This breakthrough could lead to new medications to combat childhood cancer. The study, published in Nature Communications, provides insights into how these proteins interact with each other.
A new analytical method allows scientists to track exactly where a cancer drug accumulates inside a living cell, enabling more precise treatment design. This breakthrough uses subcellular capillary sampling and laser ablation – Inductively coupled plasma – Mass spectrometry to detect trace amounts of metal in individual cells.
Researchers Dr Íris Luz Batalha and Dr Maria Shchepinova from the University of Bath have been awarded funding to test new ideas in tackling global health challenges. They will develop precision-targeted therapies for antimicrobial resistance and investigate why treatments for Type 2 diabetes don't work for everyone.
Researchers found that squalene-based adjuvants activate distinct pathways for vaccine protection and reactogenicity, including the role of IL-1β in enhancing efficacy and IL-1α in triggering local swelling. The study's findings could lead to safer vaccine design by targeting specific immune mechanisms.
Scientists at the University of Virginia Health System have developed a suite of AI-powered tools, called YuelDesign, YuelPocket and YuelBond, to transform how new drugs are created. These tools can design drug molecules tailored to fit their protein targets exactly, even accounting for protein flexibility.
Researchers have created a 3D experimental system to study the response of low-grade gliomas to drugs, providing a more realistic environment for testing treatments. The system uses organoids from pluripotent stem cells to replicate glioma development and characteristics.
Researchers at the University of Waterloo have discovered a safer and more effective way to treat Alzheimer's disease by combining anti-amyloid antibodies with molecules found in grapes, berries, peanuts, and turmeric. This combination approach neutralizes amyloid protein clumping in the brain, leading to better outcomes.
OYE-101, a novel intravenous caffeine formulation, has been developed to accelerate recovery from general anesthesia and deep sedation. The company's lead program is being evaluated in a scientific bridging study to support a 505(b)(2) NDA for OYE-101.
Researchers have created a detailed molecular map of the thromboxane A2 receptor, which could lead to the development of safer and more effective therapies targeting this clinically important signalling system. The study reveals an unusual activation switch and provides insights into how signalling molecules interact with the receptor.
The San Antonio Breast Cancer Symposium generated nearly $40 million in economic impact and drew over 11,000 attendees from 104 countries. The symposium also produced $1.5 million in local tax revenue and supported more than 6,800 jobs.
A new model of brain wiring could shorten the path to developing medicines for multiple sclerosis and other degenerative brain diseases. The model, made of a water-filled gel, replicates the physical properties of human axons and has been successfully grown from human cells in the laboratory.
A team of researchers has created a 3D model of pediatric brain tumors using biopsy-derived organoids, allowing for more accurate testing of new drugs. The model, which accurately reproduces the human environment, preserves the molecular characteristics of the original tumor and maintains cellular heterogeneity.
Researchers have discovered a new biomarker for immunoglobulin A (IgA) nephropathy, which is an autoimmune disease that can lead to kidney failure. Complement pathway proteins, including CFHR1, were found to be overexpressed in patients with IgA nephropathy, suggesting a potential treatment target and diagnostic marker.
A study reveals dermcidin, a human body natural molecule, exhibits antiviral activity against the influenza virus. Higher baseline levels of dermcidin in individuals who don't develop flu-like symptoms may be associated with lower susceptibility to infection.
Early-stage drug development worldwide has nearly doubled over the past decade, with China's share increasing from 8% to 32%, according to Georgetown University researchers. The number of early-stage programs increased by 82.4% from 10,417 in 2015 to 18,999 in 2024.
Researchers have identified a framework to characterize RNA structure-changing small molecule drugs, which could lead to the development of better treatments targeting RNA directly. The study found that molecules binding to RNA rarely affect its function, while those altering its structure have a bigger impact.
Researchers have developed a low-cost fake drug detector that uses an infrared sensor to track pill dissolution rates. The device can identify legitimate medications from counterfeit ones with high accuracy, making it a valuable tool in the fight against global health threats.
A new clinical trial will investigate whether adding the oral medication vorasidenib to standard chemotherapy improves progression-free survival for people with newly-diagnosed, grade 3 IDH-mutant astrocytoma. The study aims to recruit 400 individuals with this type of brain cancer and evaluate the safety and side-effect profile of the...
Researchers at the University of Edinburgh have developed a method to transform post-consumer plastic into L-DOPA, a frontline medication for Parkinson's disease. The process harnesses the power of bacteria to break down PET waste and produce L-DOPA, providing a sustainable solution to the production of pharmaceuticals.
ToxIndex integrates AI agents to access and orchestrate toxicological resources, providing comprehensive risk assessments in hours. The platform addresses a critical need in chemical and drug safety, meeting the vision of the 2007 NRC report for 21st-century safety testing.
ToxIndex integrates three tiers of New Approach Methodologies, leveraging AI agents to access and orchestrate toxicological resources, and providing comprehensive, source-traceable risk assessments in a fraction of the time required by traditional methods.
Researchers have identified and resolved molecular bottlenecks to produce doxorubicin, a vital chemotherapy agent, resulting in a 180% increase in production. This breakthrough enables cost-effective manufacturing of essential antibiotics and anti-cancer agents, promising a cleaner and more reliable supply of life-saving medicines.
A preclinical study reveals a new topical cream that activates the skin's immune defenses and suppresses tumor growth in two models of cutaneous squamous cell carcinoma. The cream works by blocking LSD1, an enzyme that suppresses immune-activating pathways in the skin, prompting immune cells to attack tumors.
Researchers discovered yaku'amide B induces CD9 degradation, a cancer stem cell-related protein, in addition to inhibiting ATP synthase, leading to cellular energy depletion and cancer cell suppression. This natural compound has potential as a new therapeutic approach for cancer treatment.
Researchers developed a machine-learning system that predicts how molecules form, cutting lab work time from months to days and reducing costs. The system uses asymmetric cross-coupling reactions to build complex compounds and can be applied across fields, deepening our understanding of chemistry.
The Alliance for Clinical Trials in Oncology is spotlighting new trials for colorectal cancer in March, focusing on early detection methods and treatments for treatment delays and loss of appetite. The trials aim to improve patient outcomes, with several enrolling patients with newly diagnosed colon or rectal cancer.
Researchers at Goethe University are developing non-hormonal contraceptives to address declining pill use and side effects. The PREVENT project aims to create safe and effective alternatives, focusing on small molecules that block proteins in sperm or egg cells.
Researchers have developed a bacterial system to create millions of potential drug molecules that can target difficult-to-treat cancers. The approach combines chemical peptide stabilisation with the TBS assay to screen for effective peptides, which can then be tested in more complex tissue models and animal studies.
The European Medicines Agency has granted a positive opinion to Acoziborole Winthrop as a single-dose oral treatment for both early- and advanced-stage gambiense sleeping sickness in adults and adolescents. The medicine, co-developed by DNDi and Sanofi, could provide a significant advance over current therapies.
Researchers at Osaka Metropolitan University found that targeting the glutamine transporter ASCT2 can suppress cell growth and induce apoptosis in synovial sarcoma cells. This study suggests a new approach for treating this aggressive malignant tumor by cutting off its nutrient supply.
Approximately 40 cancer patients will receive LMP744 for five consecutive days, with biological analyses conducted on brain tissues before and after treatment. If results are favorable, treatment will continue for 12 cycles to evaluate parameters such as progression-free survival and overall survival.
The project aims to test whether reducing chronic inflammation triggered by DNA can help older adults stay healthier. The research focuses on retrotransposons, which become increasingly active with age, leading to tissue decline.
A UC Santa Barbara research team has developed a method to efficiently synthesize non-natural amino acids and apply them to peptide construction. This technique provides greater access to amino acids beyond the 22 found in nature, opening up new possibilities for biochemists, medical researchers, and materials scientists.
Chemists at the University of Münster developed a new method to produce high-grade housane molecules, which are small tri- or quadripartite ring molecules crucial for drug development and materials science. The reaction is triggered by photocatalysis, enabling efficient access to valuable products.
The collaboration aims to integrate Mayo Clinic's clinical insights and genomic data with Merck's AI capabilities to support drug discovery and development. The partnership will focus on high-need therapeutic areas, leveraging multimodal data sets and advanced analytics to advance progress in disease treatment.
Researchers developed a free-to-use software tool, PSBench, to verify the accuracy of artificial intelligence-based protein structure predictions. The database includes 1.4 million annotated protein models, verified by experts, and provides reliable information for building more accurate AI systems.
Researchers are developing a new compound that can break down MDM2, a cancer-driving protein often found in triple-negative breast cancer, showing promise in shrinking tumors. The goal is to create an entirely new class of therapies for this aggressive and difficult-to-treat form of the disease.
Kobe University scientists have engineered bacteria to produce a group of compounds with promising pharmacological activities. The breakthrough uses a rational design strategy to create a platform for industrial production of drug candidates.
MIT researchers used a large language model to optimize the genetic sequences of proteins manufactured by yeast, reducing production costs. The new model predicted which codons would work best for manufacturing six different proteins, including human growth hormone and a monoclonal antibody, with successful results.
A recent study reveals that hydration and terminal chemistry of poly(ethylene) glycol (PEG) polymers influence immune recognition, providing insights into why some PEGylated drug coatings fail to evade the immune system. The findings pave the way for novel drug coatings with improved performance.
A new HPV cancer vaccine developed by Northwestern University scientists has shown promising results in a preclinical model. The vaccine's carefully organized structure dramatically enhances the immune system's ability to attack tumors, shrinking them and extending animal survival.