Articles tagged with Drug Development
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.
A five-year NIH-funded study will explore the role of glucagon hormone in human metabolism, focusing on its impact on insulin secretion, glucose regulation, and energy balance. The research aims to clarify glucagon's function and inform more effective drug development for obesity and Type 2 diabetes treatment.
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.
The Virtual Cell Pharmacology Initiative (VCPI) aims to build the first standardized framework for virtual cell modeling in drug discovery. Ginkgo Datapoints is offering free high-throughput RNA profiling via its platform, generating over 12 billion data points and aiming to test at least 100,000 compounds.
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 ...
A team at the University of Pennsylvania has solved the mechanism of action of hydralazine, revealing its potential to halt the growth of brain cancer cells. By blocking an oxygen-sensing enzyme, hydralazine can reduce intracellular calcium levels, causing blood vessels to relax and tumor cells to enter a dormant state.
Clinical trial participants are calling for more inclusive and patient-centered cancer research, highlighting the need for transparent designs and improved communication. The authors propose various improvements to trial design and delivery, including expanding eligibility criteria and involving patient advocates in trial design.
Researchers found that CRISPR-Cas9 gene editing persists longer and produces more predictable results in non-dividing neurons. They also discovered new DNA repair genes that can be used to control gene editing outcomes, which could lead to safer and more effective therapies for genetic diseases.
A new computational tool called DeepTarget predicts direct and indirect targets of cancer drugs, revealing that small molecules can have different targets and effects depending on the disease and cell type. The study demonstrates the tool's superior performance in real-world scenarios, highlighting its potential to accelerate drug deve...
A new compound MCH11, a monoacylglycerol lipase inhibitor, shows promising effects in reducing alcohol consumption and motivation to drink in mice. The molecule exhibits anxiolytic and antidepressant properties with sex-dependent efficacy, correcting genetic alterations associated with alcohol use disorder.
Researchers at the University of Kansas have discovered a human gene, PARP14, that plays a role in regulating interferon and boosting the immune response. The protein's antiviral activity against multiple viruses suggests it could guide the development of new therapies for viral infections.
Stuart S. Martin, PhD, has been appointed Chair of the Department of Pharmacology & Physiology at UMSOM, bringing over 20 years of success in advancing basic science research efforts and developing new drug therapies. His groundbreaking discovery of microtentacles on cancer cells promises to lead to new life-extending therapies.
Advanced molecular dynamics simulations model complex RNA structures with high accuracy, enabling potential applications in RNA-based therapies and drug design. The study successfully simulated the folding of diverse RNA stem loops, revealing a distinct folding pathway for challenging motifs.
Scientists at Wake Forest University School of Medicine have discovered a new way to kill cancer cells by blocking their ability to clean up harmful waste. By inhibiting peroxiredoxin-3, researchers found that toxic levels of hydrogen peroxide overwhelm cancer cells, destroying them.
A new broad-spectrum antivenom developed by DTU researchers covers 17 African snake species and provides better protection against tissue damage, with a lower risk of immune reactions. The antivenom has shown impressive results in laboratory studies and could revolutionize the treatment of venomous snakebites in Africa.
A new small molecule drug, RAGE406R, has been developed to disrupt a key cellular pathway responsible for chronic inflammation and associated complications in patients with diabetes. The breakthrough could offer a new therapeutic option for stopping the harmful effects of both type 1 and type 2 diabetes at the source.
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.
AI accelerates discovery and validation of novel therapeutic targets by integrating multi-omics data and advanced computational models, reducing the traditional decade-long research and development cycle to two to three years. AI also enhances drug efficacy, predicts resistance mechanisms and improves clinical translation.
Researchers developed a machine learning-based workflow, SPaDe-CSP, to predict crystal structures of organic molecules. The workflow narrows the search space by predicting probable space groups and crystal densities before computationally intensive relaxation steps.
Researchers from The University of Osaka and The University of Tokyo have developed a novel technology that visualizes specific molecules inside living cells using light. The new photo-responsive alkyne tag enables precise visualization without disrupting molecular dynamics.
The FDA has approved elinzanetant for treating hot flashes and night sweats in postmenopausal women. The drug significantly reduces the frequency and severity of symptoms while improving sleep quality and quality of life.
A novel therapeutic candidate has been developed to treat severe cutaneous adverse reactions such as Stevens-Johnson syndrome (SJS) and toxic epidermal necrolysis (TEN). The compound suppresses disease-specific keratinocyte death, demonstrating its efficacy in preclinical studies.
Researchers at Gladstone Institutes discover a gene called HMGN1 that disrupts DNA packaging and regulation, leading to heart malformations in people with Down syndrome. Removing the extra copy of HMGN1 from mice with Down syndrome prevents heart defects, paving the way for potential treatments.
Researchers at Sanford Burnham Prebys discovered a new mechanism to confer signaling bias in predictable ways, permitting rational design of new drugs. This breakthrough could lead to better therapies for addiction and psychiatric disorders by targeting the neurotensin receptor 1 (NTSR1) with biased modulators.
Researchers have discovered an enzyme called PapB that can create tight rings in therapeutic peptides, enabling the creation of stronger, longer-lasting versions of GLP-1 medications. This enzymatic method offers a simpler alternative to traditional chemical methods and could improve the stability and effectiveness of these drugs.
A team of UNLV researchers has engineered a new class of cannabidiol (CBD)-like medicines that show powerful seizure-reducing effects. The caraway-seed derived therapies offer a safer and more effective treatment for childhood seizure disorders than existing frontline therapies.
The ISSCR 2026 Annual Meeting will bring together academic and industry leaders to explore advances in stem cell science and regenerative medicine. Scientists can submit abstracts by February 25, 2026, for oral presentations and Travel and Merit Awards.
Organoids are transforming biomedical research with their ability to model complex diseases like cancer, Zika virus infection, and cystic fibrosis. They enable high-throughput drug testing, personalized treatment prediction, and safety assessment.
The Critical Path Institute has been recognized for its innovative approaches to regulatory science, which have measurably improved public health. The organization's work in creating practical tools and cross-sector collaborations has shortened drug development timelines and improved regulatory decisions.
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.
Isha Jain wins NIH Transformative Research Award to develop new targeted treatments using modern science and techniques, potentially leading to personalized vitamin-based therapies for genetic disorders. Her lab has made key discoveries on oxygen levels and tissue damage, aiming to fine-tune these processes for disease treatment.
A new study reveals that stevioside can effectively promote hair follicles to enter the growth phase when combined with minoxidil. The natural sweetener enhances skin absorption of the drug, leading to new hair development in a mouse model of alopecia.
A University of Houston professor has received funding to develop effective treatments for Cryptosporidium infections, which cause severe diarrhea and have no existing cure. The team aims to design drugs that target the parasite's enzyme CDPK1 to minimize systemic exposure and maximize efficacy.
A new MOF, APF-80, enables the crystalline sponge method to capture and analyze nucleophilic compounds like alkaloids. This development opens possibilities for structural analysis in drug development and biochemistry.
A new survey found that 72% of respondents agree that plasma-derived medicines can save lives, but most haven't donated. Plasma is essential in producing treatments for various serious health conditions.
A historic campaign in Argentina developed antivenom for black widow spider bites, leading to a model for pharmaceutical autonomy. This approach is crucial for fighting neglected tropical diseases globally.
Researchers at the University of Bath develop a peptide fragment that locks alpha-synuclein into its healthy shape, blocking toxic clumps that cause nerve cell death. The breakthrough demonstrates the potential of rational peptide design to transform large proteins into compact drug-like molecules.
Scientists successfully derive and maintain self-renewing and pluripotent ESCs from chickens and seven other bird species using a growing medium of egg yolk. The study holds promise for applications in studying embryonic development, producing lab-grown poultry, and reviving endangered birds.
A new study finds that over 50% of small-molecule drug patents this century are connected to NIH-backed research that would likely be cut under a 40% budget reduction. This highlights the significant impact of federally funded research on the development of life-changing medicines.
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.
Armida Labs will use the funding to advance preclinical studies of Targefrin, a potential clinical candidate for pancreatic and other cancers. The grant aims to develop an anti-metastatic agent that targets the EphA2 protein, which drives cancer cell invasion and metastasis.
Researchers at the University of Maine Forest Bioproducts Research Institute have discovered a sustainable method to produce (S)-3-hydroxy-γ-butyrolactone, a crucial building block in pharmaceuticals. This approach could significantly reduce greenhouse gas emissions and production costs by up to 60%.
The Alliance for Clinical Trials in Oncology will host a public webinar showcasing key findings from the 2025 ASCO Annual Meeting. Researchers will discuss latest information on colorectal, squamous cell, and renal cell cancers.
A new white paper from the USC Schaeffer Center recommends FDA reforms to streamline clinical trials, provide clearer guidance on newer technologies, and improve agency efficiency. This could reduce development time and costs, pushing down prices and improving patient access.
A new study from Mizzou's College of Veterinary Medicine analyzed the effects of radioactive iodine therapy on thyroid cancer in dogs. The research found that tailoring the dose of radiation more precisely for each dog could improve outcomes and potentially lead to more targeted care.
A novel drug target, CD300a, has been identified to suppress immune cell activation and prevent chronic heart and kidney failure after acute tissue injury. The treatment preserves cardiac function and reduces renal fibrosis in mice with genetic deficiency of CD300a.
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.
Researchers propose idiopathic pulmonary fibrosis as a disease-specific treatment to test general geroprotector therapies, leveraging its high overlap with aging biology. This approach could accelerate drug development and reduce costs by targeting shared biological pathways.
USC Stem Cell scientists have developed a blueprint for generating specific kidney cell types on demand, holding immense value for preclinical studies of new therapeutics and congenital kidney diseases. The team successfully created lab-grown proximal tubule cells that can absorb sugar and protein, respond to chemotherapy drugs, and pr...
The C-Path Translational Therapeutics Accelerator has completed a record year of funding and mentorship, supporting seven projects in 2025 with approximately $2.48 million in awards. The program awarded grants to teams advancing treatments for rare liver disease, drug-resistant lung infections and pediatric brain tumors.
Researchers have identified a new class of compounds derived from psychedelics that could open the door to safe, targeted therapies for widespread use in clinical settings. These PIPI drugs may modulate the immune system to control inflammation, potentially treating conditions such as Alzheimer's and Parkinson's disease.
A synthetic molecule called Pep19 has been shown to reduce visceral fat and improve sleep quality in overweight adults, with no side effects observed. The study involved 24 volunteers who took either a placebo or Pep19 in capsules, and found a 17% reduction in visceral fat and improved sleep quality in those who received the molecule.
Scientists at CUNY ASRC develop novel synthetic carbohydrate receptors that block infection from seven different viruses across five unrelated families, including Ebola and SARS-CoV-2. The breakthrough offers a promising path toward the development of broad-spectrum antivirals.
A new study offers a powerful AI-assisted method for uncovering exactly how TB drugs kill the bacteria, which could lead to smarter treatment combinations. The approach uncovers molecular details of drug interactions and can predict their impact from images alone.
Researchers found that a faster rate of binding does not always translate to greater potency, as the study's lead author David Heppner explained. The team proposed a two-step design process balancing inactivation efficiency rates with target selectivity and other parameters to identify promising compounds.
Climate-related disasters pose significant disruptions to US drug manufacturing facilities, affecting nearly two-thirds of production sites. Researchers assessed the impact of disaster events on counties with US drug production facilities and found that nearly two-thirds were located in areas affected by at least one disaster declaration.
The new model can predict how well any given molecule will dissolve in an organic solvent, helping chemists choose the right solvent for reactions. The researchers trained two models on a comprehensive dataset and found that their predictions were two to three times more accurate than the previous best model.
MIT researchers have developed a method to reveal the inner workings of protein language models, which can accurately predict proteins suitable for drug or vaccine targets. By analyzing sparse representations of proteins, they identified key features that drive these predictions.
Researchers develop novel treatment for acetaminophen-induced liver injury (AILI) using a GSDMD inhibitor. YM81 selectively binds to and inhibits the pyroptosis protein gasdermin D, reducing liver inflammation and damage.
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.