Articles tagged with Drug Candidates
A large-scale Phase 3 trial of CRISPR therapy has shown an 87% reduction in attacks for patients with hereditary angioedema. The treatment also improved quality-of-life scores and reduced the need for on-demand medication, paving the way for future genetic therapies.
Two projects, BOOST-HP and BIONIC, use machine learning to analyze large datasets of medication exposure and outcomes. The findings highlight the need for more data and cautious design of machine learning models to accurately assess potential links between medications and pregnant women.
Researchers have developed a potent and selective small-molecule inverse agonist targeting the itch receptor MRGPRX4, providing a new strategy for treating cholestatic itch. The compound HEP-50768 showed efficacy in animal studies and demonstrated good drug-like properties and safety profiles.
A recent NIH-funded study reveals that oral small-molecule GLP-1 drugs can suppress eating for pleasure and reduce cravings by targeting a deeper brain region, potentially treating other dysfunctions in reward processing. The findings provide insight into the neural mechanisms underlying the effects of these medications.
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...
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.
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.
A new Cochrane review of 17 clinical trials found that anti-amyloid Alzheimer's drugs have no significant impact on cognitive decline or dementia severity, but may increase the risk of brain swelling and bleeding. The evidence suggests that these drugs are unlikely to provide clear benefit to patients.
Researchers found that clonal hematopoiesis accelerates aortic aneurysm progression and that commonly used osteoporosis drugs can slow or halt this process. The study identified a key mechanism by which macrophages contribute to extracellular matrix degradation, providing a potential non-surgical approach for treating aortic aneurysms.
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.
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.
A hormone called FGF21 reverses obesity in mice by signaling to the hindbrain, a region targeted by GLP-1 drugs. This finding provides insight into the naturally occurring hormone's benefits for weight loss and MASH treatment.
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.
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.
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.
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.
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.
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.
OYE-101, a novel intravenous caffeine formulation, aims to accelerate recovery from general anesthesia and deep sedation in adults. The FDA-cleared treatment also seeks to address opioid-induced respiratory depression and related overdose situations.
A Purdue University team led by Kyle Cottrell has discovered a new therapeutic target for triple-negative breast cancer, a deadly form of breast cancer lacking targeted therapies. The researchers identified dsRNA-binding proteins, specifically PACT, which suppress another protein called RNA-activated protein kinase (PKR).
Researchers at University Hospitals and Case Western Reserve University have discovered a novel enzyme, SCoR2, that removes nitric oxide from proteins controlling fat build-up. Inhibition of this enzyme prevents weight gain and liver injury in mouse models, also lowering bad cholesterol.
In a new study, Northwestern scientists identified a previously unknown toxic sub-species of amyloid beta oligomers that drive brain changes in Alzheimer's disease. NU-9 decreased this toxin and reduced damage in a mouse model, suggesting it could prevent or delay the cascade of toxic events that destroy neurons.
A new study demonstrates a one-two punch approach to combatting drug-resistant tuberculosis by pairing rifampicin with AAP-SO2, which exploits the weaknesses of resistant bacteria. The combination effectively kills more bacteria than either drug alone, increasing potency and reducing resistance.
Researchers at Martin Luther University Halle-Wittenberg have developed a promising new substance that inhibits the ability of tuberculosis bacteria to produce energy and causes them to die. The compound, PRP020, targets the pathogen's ATP synthase but attacks a different site than existing drugs like bedaquiline.
Biomedical researchers recommend diversifying funding sources, pursuing earlier licensing and commercialization, and fostering international collaborations. The US drug discovery landscape is at risk due to federal funding cuts, and alternative approaches are needed to ensure continued progress.
Researchers at the University of Virginia Health System have developed a new treatment for acute myeloid leukemia, a deadly form of blood cancer. The FDA-approved medication works by disrupting cellular protein interactions that drive leukemia cell growth and survival, offering patients a potential cure.
Researchers develop ex vivo treatment of blood stem cells with Rhosin, a RhoA inhibitor, to rejuvenate them and improve the production of healthy blood cells. This strategy targets the core of the ageing process, making blood stem cells more capable of regenerating and producing new healthy blood cells.
The article highlights the mismatch between psychedelics and economic drug development principles. Pharmaceutical companies are developing short-acting compounds and neuroplastogens to engineer trips out of the experience altogether. Dr. Sandy Hager's research suggests investors should remain cautious due to weak intellectual property ...
Researchers found that AI models predict protein structures despite modifications in amino acid sequences or ligands, indicating a lack of understanding of physical chemistry. The models only recognize patterns they've seen before and struggle with unknown proteins.
Researchers at Helmholtz Centre for Infection Research have identified a new drug candidate, Substance 31, that prevents the production of new proteins in malaria parasites. This approach has the potential to break through existing resistances and develop new therapeutic strategies against malaria infections.
A team of scientists at HIPS has developed a chemo-enzymatic platform to produce large quantities of furanolides, a class of natural products with high structural diversity. The resulting substances have shown promising biological activities against bacterial pathogens and cancer cells.
Denis Evseenko and Toby Maher are developing a regenerative drug to block cells that promote fibrosis in the lungs, aiming to slow or reverse IPF damage. The team plans to test the safety and therapeutic potential of their drug-like molecules in animals and human cells.
A large international clinical trial found that elinzanetant significantly reduces hot flashes and night sweats in postmenopausal women by over 73%. The drug also shows secondary benefits such as improved quality of life and reduced sleep disturbances, with no harmful effects on the liver or bone density.
Ibezapolstat, a new antibiotic, has been shown to be effective in treating Clostridioides difficile (C. diff) infections with high rates of sustained clinical cures. The study found that ibezapolstat killed harmful bacteria without harming the good bacteria in the gut, which helps prevent recurrent C. diff infections.
A study in mice found that high triglyceride levels directly cause abdominal aortic aneurysms, challenging the notion that they are merely biomarkers of vascular disease. Researchers identified specific proteins and lipoproteins as causal drivers of aneurysm development and growth.
The 2025 Critical Path Institute Global Impact Conference will bring together industry, regulatory agencies, academia, and patient advocacy groups to discuss pressing challenges in drug development. The event aims to foster cross-sector collaboration to advance medical innovation and improve treatment outcomes for patients.
The European Research Council has awarded three ERC Proof of Concept grants to Göttingen University professors, enabling the development of initiatives that can benefit Europe's economy and society. The projects focus on harnessing renewable energy, reducing chemical waste, and improving biomedical image analysis.
Scientists from the University of Bath have identified two new families of chemical compounds that inhibit alpha-methylacyl-CoA racemase (MCR) in Mycobacterium tuberculosis, a key enzyme for TB survival. This breakthrough could lead to new treatments for TB and potentially other diseases like prostate cancer.
UVA Health received two anonymous $25 million estate gifts to support the Paul and Diane Manning Institute of Biotechnology. The institute will develop new treatments for hard-to-treat diseases with a state-of-the-art biomedical research facility expected to drive economic growth in Central Virginia.
Scientists have discovered a novel way to block an enzyme involved in regulating blood pressure, called ACE. Ciprofloxacin binds selectively to a different site, blocking angiotensin I but not inhibiting the enzyme's other functions.
The research aims to improve the survivability of wounded soldiers, enhance combat safety, and accelerate postoperative recovery in a multicenter pivotal clinical study. OYE's technology has shown promise in reducing mortality rates, lowering healthcare costs, and improving surgical workflow efficiency.
Researchers found that an inexpensive HIV drug can improve vision in patients with diabetic macular edema (DME) more effectively and at a lower cost than existing treatments. The drug, lamivudine, is taken orally and may represent a game-changing option for millions of patients worldwide.
The Center for Research Innovation in Biotechnology (CRIB) has developed a comprehensive database of active pharmaceutical ingredients with evidence of clinical testing. The database provides valuable information on drug discovery and development, including pricing, sponsors, and intended clinical applications.
Researchers at UCLA Health identified a candidate small molecule, DDL-357, that increases concentrations of secreted clusterin, reducing toxic protein phospho-tau and improving mitochondrial function. The drug also improved memory in treated mice in maze-based cognitive tests.
A team of researchers has discovered a small molecule that can selectively block cell death, which could lead to new treatments for neurodegenerative conditions. By targeting the killer protein BAX, the molecule can prevent excessive cell death in neurons, potentially slowing or halting disease progression.
Researchers have developed a new compound, SHP1705, that selectively attacks glioblastoma stem cells by hijacked circadian clock proteins. The compound was found to be safe and well-tolerated in humans during a phase 1 clinical trial.
A new generative AI model, DiffSMol, has been developed to generate realistic 3D structures of small molecules with promising drug properties. The study achieved a 61.4% success rate in creating novel molecules, outperforming prior research attempts.
Researchers at UCSF's AViDD Center have developed a highly bioavailable, covalent molecule that inhibits coronavirus MPro with potent in vivo efficacy. This discovery holds promise for preventing future coronavirus pandemics.
Researchers successfully treated atherosclerosis and fatty liver disease using DT-109 in nonhuman primates, which has potential as a dual therapy for two common conditions. The compound reduced the formation of atherosclerotic plaques and stopped critical processes that lead to vascular calcification.
Insilico Medicine is presenting significant achievements in cancer therapy and immunology, including AI-powered discovery of potential therapies and novel solutions for unmet needs. The company's Pharma.AI platform has received IND clearance for 10 molecules, with promising results in Phase 2a clinical studies.
A new class of agents has been developed to inhibit hospital germs, particularly targeting the toxin α-hemolysin produced by Staphylococcus aureus. The compound H052 showed high effectiveness in cell culture and animal models, increasing survival rates in infected mice.
Researchers have developed a new approach to personalize treatments for young cancer patients by growing tumors in chicken eggs and analyzing proteins. The technique, which combines genomics and proteomics, was successful in identifying a treatment option for a patient with a rare pediatric cancer.
Researchers developed new AI models, InstaNovo and InstaNovo+, to vastly improve accuracy and discovery in protein science. These models excel in tasks such as de novo peptide sequencing, identifying microorganisms, and discovering novel peptides, with implications for personalized medicine, cancer immunology, and beyond.
Scientists have discovered peptides that bind irreversibly to the transcription factor cJun, permanently blocking its action in cancer cells. The study uses a new screening platform technology and has shown promising results for developing new cancer treatments.
Researchers at the University of Virginia Health System have developed a computational tool called LogiRx that can predict how drugs will affect biological processes in the body. The tool has demonstrated its potential by identifying a promising candidate to prevent heart failure, a leading cause of death worldwide.