All Small Molecules articles
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NewsAI and genetics identify WNK2 inhibitor for osteoarthritis treatment
Researchers at University of Utah Health have used artificial intelligence and human genetic data to identify a promising early-stage drug candidate targeting WNK2, a protein linked to inherited osteoarthritis, with the lead compound reducing inflammatory gene activity and supporting cartilage cell health in laboratory models.
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NewsNew molecular glue platform broadens E3 ligase targeting for cancer drug development
Researchers at Dana-Farber Cancer Institute have developed a scalable platform for discovering molecular glue degraders, reporting the first metabolically activated example and broadening the scope of targeted protein degradation in cancer.
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NewsTargeting the roots of head and neck cancer: Why MDA-9 matters
A small molecule inhibitor developed at Virginia Commonwealth University has demonstrated the ability to eliminate cancer stem cells and reverse cisplatin resistance in preclinical models of head and neck squamous cell carcinoma, offering a potential new strategy to address one of oncology’s most persistent clinical challenges.
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NewsTriple drug combination targets cancer and senescent cells simultaneously
A three-drug combination targeting shared metabolic vulnerabilities in cancer and senescent cells has improved physical performance and extended survival in aged mice, whilst avoiding the thrombocytopenia associated with existing navitoclax-based regimens.
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NewsOral antiviral GHP-88310 candidate shown to block measles transmission in ferret model
An experimental oral antiviral has demonstrated the ability to prevent both airborne and contact-based transmission of a measles surrogate virus in ferrets, offering a potential new tool to complement vaccination during outbreaks.
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NewsInsilico Medicine nominates ISM9528 non-opioid chronic pain candidate using AI
Insilico Medicine has nominated ISM9528, an orally available, brain-penetrant non-opioid candidate targeting a previously unrecognised pain mechanism, as its 31st AI-generated preclinical compound since 2021, with efficacy exceeding morphine in some preclinical models.
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NewsNew drug SB-0110 targets levodopa’s two biggest clinical limitations for Parkinson’s
A computational biology-derived compound has shown promise in preclinical models by simultaneously improving levodopa response and reducing dyskinesia — two of the most significant challenges in long-term Parkinson’s disease management.
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NewsNTX-301 targets treatment-resistant AML via Hippo pathway in preclinical study
A novel epigenetic therapy has demonstrated activity against treatment-resistant acute myeloid leukaemia in preclinical models, including high-risk TP53-mutant disease, by selectively reactivating the Hippo tumour-suppressor pathway.
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NewsNew BET therapies could help beat resistance in solid cancers
A new review finds that next-generation BET-targeted therapies, including selective inhibitors and PROTACs, are addressing the clinical shortcomings of earlier compounds and reviving interest in this approach for solid tumour treatment.
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NewsAUTAC strategy targets MCL1 to overcome myeloma treatment resistance
Scientists at VCU Massey Comprehensive Cancer Center have developed an autophagy-targeting chimera that redirects cancer cells’ own recycling machinery to degrade the survival protein MCL1, offering a potential new strategy to overcome treatment resistance in multiple myeloma.
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NewsNew BET inhibitors could overcome barriers in solid tumour treatment
A new review examines how advances in drug design, PROTAC degraders and combination therapies are reviving the clinical prospects of BET inhibition in solid tumours, after early-generation compounds were hampered by toxicity, resistance and modest efficacy.
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NewsKCL-286 targets multiple Alzheimer’s pathways in preclinical study
A repurposed small molecule originally developed for spinal cord injury has demonstrated the ability to address multiple disease-relevant pathways in Alzheimer’s disease, including DNA damage and neuroinflammation, offering a potential route to disease-modifying therapy.
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NewsInsilico Medicine launches Phase III trial of AI-designed Rentosertib drug
Insilico Medicine has advanced its AI-discovered TNIK inhibitor Rentosertib into a 320-patient Phase III trial for idiopathic pulmonary fibrosis, marking a landmark moment for generative AI drug discovery.
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NewsAI and lab techniques accelerate tuberculosis drug discovery
Researchers at UMass Amherst have combined high-throughput laboratory screening with an AI neural network to identify compounds capable of breaching the protective outer membrane of Mycobacterium tuberculosis, potentially accelerating the search for new TB therapeutics.
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NewsXL20 drug candidate shields motor neurons from ALS-linked TDP-43 damage
A newly identified experimental compound, XL20, has demonstrated neuroprotective effects in mouse models and human motor neurons by targeting a conserved disease-linked region of TDP-43, a protein central to ALS pathology.
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NewsHidden vulnerability in persister cells could prevent KRAS cancer relapse
Scientists at Chiba University have identified a metabolic vulnerability in drug-tolerant cancer cells that survive KRAS-targeted therapy, opening a potential route to combination treatments designed to prevent disease recurrence in lung, pancreatic and colorectal cancers.
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NewsLargest chemical reactions database launched to boost AI drug discovery
Researchers at the University of Michigan have assembled a database of more than 50,000 chemical experiments, offering AI systems an unprecedented resource to accelerate drug discovery and reduce reliance on scarce precious metal catalysts.
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NewsGinkgo Bioworks launches ADME-One platform for early-stage screening
Ginkgo Bioworks has launched ADME-One, an integrated platform combining high-throughput ADME testing with AI-powered human pharmacokinetic projections.
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NewsNew malaria drug T111 targets all parasite stages in single treatment
A novel compound developed over 15 years of research has demonstrated unprecedented activity against liver, blood and sexual stages of Plasmodium parasites, possibly positioning it as a potential cure for malaria.
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NewsSafer diazo compound synthesis advances medicinal chemistry capabilities
Researchers at Tokyo University of Science have developed a safer synthetic route to β-heteroatom-substituted diazo esters, eliminating the need for toxic diazomethane. The azide-to-diazo conversion method uses mild conditions and readily available starting materials, potentially streamlining early-stage drug discovery and compound library synthesis.


