Articles tagged with Drug Discovery
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.
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.
A new large language model, LassoESM, has been developed to predict lasso peptide properties, enabling the acceleration of rational design for biomedical applications. The model was trained on thousands of lasso peptide sequences and demonstrated accurate prediction of various properties.
Dr. Benjamin P. Brown proposes a targeted approach to improve the accuracy and speed of machine learning in drug discovery. His work focuses on creating a more generalizable deep learning framework for structure-based protein-ligand affinity ranking.
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 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.
The study identified chemical compounds that precisely block the interaction between RAS and a key pathway for tumour growth. The treatment has entered its first clinical trial in humans and has shown promising results, with potential to treat many different types of cancers while avoiding effects on healthy cells.
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.
Pharma.AI is a comprehensive generative AI-powered drug discovery platform featuring PandaOmics, Generative Biologics, Chemistry42, Science42: DORA, and PreciousGPT. These platforms enhance target and biomarker discovery, biologics engineering, small molecule design, scientific research, and disease modelling.
Researchers identified citronellyl butyrate and other compounds with high binding affinity to DPP-4, suggesting strong inhibitory potential. The compounds' favorable pharmacokinetic properties, such as high absorption and blood-brain barrier permeability, make them promising candidates for antidiabetic treatments.
Researchers identify three primary UPR pathways and their downstream cascades, which play a crucial role in the differentiation of osteoblasts and osteoclasts. Targeting these pathways with emerging drugs may alleviate bone-related events and kill tumors localized in bones.
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.
The NF-κB signaling pathway plays a key role in regulating immune responses, inflammation, cell development, and proliferation. Research into its functions and mechanisms continues to uncover new breakthroughs in immunology and life sciences.
Researchers discovered onnamides, a family of compounds from Okinawan marine sponges, showing remarkable potential against Leishmania major. These compounds demonstrate potency and safety exceeding current treatments, offering new approaches to overcome drug resistance.
The USC team is developing a new drug aimed at a previously unexplored biological target in Alzheimer’s disease, focusing on a trigger of brain inflammation. They have identified an enzyme that predisposes the brain to inflammation and are now working to find a drug that can block it without harmful side effects.
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.
A novel method combining biological experiments and information science techniques reveals cancer cells' preference for aerobic glycolysis despite sufficient oxygen availability. This research provides a powerful tool for identifying metabolic vulnerabilities in cancer cells, which could lead to more effective treatments.
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.
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.
The global market for AI in pharmaceuticals is projected to reach $13.4 billion by 2035, driven by Insilico Medicine's innovative use of generative AI. The company will share its achievements at a satellite forum during BIOHK2025, showcasing significant efficiency boosts compared to traditional methods.
The latest issue of SLAS Technology highlights significant advancements in biomedicine and diagnostics, with AI-powered tools achieving 99.9% accuracy in detecting monkeypox. Additionally, the journal showcases innovative lab technologies, including multi-camera zebrafish assays and infection-proof titanium implants.
Researchers at the University of Pennsylvania used AI to identify previously unknown compounds in Archaea that could fuel the development of next-generation antibiotics. The study, published in Nature Microbiology, found that 93% of the identified archaeasins demonstrated antimicrobial activity against drug-resistant bacteria.
The new book explores organoid bioengineering, using pluripotent and adult stem cells to create models for various organs. It discusses breakthroughs in personalized cancer care and large-scale production protocols supported by advanced tools like 3D printing and genome editing.
The journal features novel FAK-paxillin inhibitors, a venom toxin screening platform, and AI-driven solubility prediction for compound discovery. SLAS Discovery highlights innovative technologies to understand and treat human disease.
Repair Biotechnologies develops therapies targeting toxic excess free cholesterol in cells, a hallmark of aging and obesity. The company is advancing a lead drug for accelerated atherosclerosis, with ARDD providing opportunities to connect with brilliant minds and showcase groundbreaking research.
LongGame Ventures supports cutting-edge research in longevity biotechnology at ARDD 2025, featuring Nobel laureates and industry leaders. The conference aims to advance healthspan and longevity science, with a focus on regenerative medicine and cellular replacement.
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 inaugural ESMO Targeted Anticancer Therapies (TAT) Asia Congress 2025 will convene a diverse group of stakeholders to foster collaboration and accelerate the development of innovative cancer therapies. The congress aims to become a catalyst for global collaboration and innovation in oncology.
The Society for Laboratory Automation and Screening's two scientific journals, SLAS Discovery and SLAS Technology, have achieved substantial impact factor increases due to open access publishing. This shift has led to higher citations and visibility for authors, with SLAS Technology experiencing a notable rise of 1.2 points to 3.7.
Scientists have identified compounds that can help cells fend off viral infection by activating the integrated stress response pathway. These compounds show promise as broad-spectrum antivirals against multiple types of viruses and have been tested in human cells and a mouse model with positive results.
Two Bar-Ilan University researchers have won ERC grants to advance cancer treatments using targeted protein degradation and improve regulatory effectiveness through machine learning. The projects aim to pioneer new therapeutics and design more effective interventions based on empirical evidence.
At the upcoming AI for Good Global Summit, Alex Zhavoronkov PhD will discuss the future potential of generative AI. Insilico Medicine's founder and CEO has invented critical technologies in generative AI and reinforcement learning, and his keynote will explore applications in drug discovery and longevity.
A team of researchers proposes a novel adaptive platform trial fund to accelerate ALS drug development. The study outlines a financing model that merges efficiencies with an innovative royalty-based investment structure, drawing on data from the HEALEY ALS Platform Trial. This new approach could transform how diseases like ALS are addr...
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.
Researchers at Florida Atlantic University have secured two key grants to investigate targeting the MBLAC1 gene as a new approach to treat glioblastoma, a very aggressive and fast-growing type of brain cancer. The project aims to advance innovative projects that could make a meaningful impact on cancer therapy.
A new computational tool called ColdBrew predicts the likelihood of water molecule positions within experimental protein structures, aiding drug discovery efforts. The tool leverages data on extensive protein water networks, providing insights into how ligands bind to proteins and potentially improving binding sites.
This issue of SLAS Technology features a high-precision microfluidic flow splitter that outperforms commercial alternatives, enabling even flow division and simplifying multi-inlet perfusion. The journal showcases technological leaps in the life sciences, including rapid pathogen detection and AI-driven insights into schizophrenia.
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.
Researchers tackle pressing challenges in drug discovery with innovative approaches, including high-throughput TRIP13 inhibitors and tau aggregation blockers. The journal focuses on advancing life sciences discovery and technology via education, knowledge exchange and global community building.
Researchers at the University of Pennsylvania School of Engineering and Applied Science have turned a deadly fungus into a potent cancer-fighting compound. The new compound, called asperigimycins, has shown promising results against leukemia cells, rivaling FDA-approved drugs.
Critical Path Institute's Translational Therapeutics Accelerator awards $250,000 to researchers advancing a promising therapeutic pathway for type 1 diabetes. The project aims to address key challenges in T1D treatment, including beta-cell preservation and immune system modulation.
A new study reveals that Rapamycin has a comparable life-extending effect to dietary restriction in eight species of vertebrates. The compound, originally developed as an immunosuppressant, offers similar benefits without the need for caloric restriction.
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 Lieber Institute is developing a new tool called GRAPE that combines generative and predictive AI to find new, more effective treatments for brain disorders such as schizophrenia. The institute is utilizing AWS gen AI and compute services to advance research and store its massive collection of genomic and other data in the cloud.
The Human Exposome Moonshot initiative aims to map the physical, chemical, biological and psychosocial exposures driving 80% of chronic diseases. The exposome project integrates advanced technologies to create a comprehensive understanding of environmental influences on health.
A team of researchers used AI and human collaboration to identify six promising drug pairs against breast cancer cells. The combinations, including simvastatin and disulfiram, showed potential for further research in therapeutic repurposing.
The Phase IIa trial of Rentosertib showed encouraging clinical data, with patients receiving the treatment experiencing significant improvements in lung function compared to placebo. The study also validated the biological mechanism of TNIK inhibition using exploratory biomarkers analyses.
A new study suggests that a drug used to prevent alcohol abuse can also interrupt runaway cell death and inflammation triggered by severe trauma, particularly in female mice. The findings may lead to therapies that could shorten hospital stays and improve survival rates if administered promptly after traumatic injuries.
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.
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.
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.
The Human Exposome is a global scientific effort to understand the environmental factors that underpin disease and health. The Exposome Moonshot Forum aims to chart this exposome, providing usable metrics and data points for targeted public health interventions.
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.
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.
Insilico Medicine will present detailed Phase IIa data on rentosertib, a novel TNIK inhibitor developed using generative AI, for the treatment of idiopathic pulmonary fibrosis (IPF). The company has previously demonstrated encouraging results from its Phase IIa study, showing favorable safety and tolerability across all dose levels.
University of Oklahoma researchers have developed a method to add a single carbon atom to drug molecules, increasing chemical diversity without compromising sensitive structures. This technique, called skeletal editing, has the potential to revolutionize DNA-encoded library technology and reduce healthcare costs.
The Psychedelics journal has expanded its focus to include all psychoactive drugs, challenging traditional classifications and embracing a broader understanding of consciousness-altering substances. This move aims to reveal novel therapeutic applications and deepen human knowledge of the mind.
Andrew Yang will study proteins crossing the blood-brain barrier and their role in brain health, seeking new therapies for neurological diseases. The 2025 Searle Scholar's research could lead to a better understanding of age-related disorders.
Researchers at Carnegie Mellon University have developed a novel FRESH bioprinting technique that enables the creation of microphysiologic systems entirely out of collagen, cells, and other proteins. This advancement expands the capabilities of studying disease and building tissues for therapy, such as Type 1 diabetes.