All Drug Targets articles
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News3D genome reorganisation identified as Alzheimer’s disease molecular hallmark
An international study combining single-cell genomics, spatial transcriptomics and artificial intelligence has identified large-scale changes in three-dimensional genome organisation in Alzheimer’s-affected brain cells, establishing chromatin architecture as a previously overlooked component of the disease’s molecular pathology and a potential framework for future therapeutic targeting.
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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 target identified to protect hearts in DMD patients
Researchers at the University of South Florida have identified NOX4 as a potential therapeutic target for DMD-related cardiomyopathy, with experimental drug Setanaxib demonstrating cardioprotective effects in preclinical models.
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WebinarMeeting ethical & regulatory requirements when using human biological specimens
Whether you source, purchase or work with human biospecimens, understanding today’s ethical and regulatory expectations is essential. Join this webinar to explore informed consent, privacy and the principles that support responsible research.
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ArticleHow AI and human biology data led to a new addiction drug candidate
How can human biology data improve target selection? Learn how one discovery programme identified a potential new treatment for opioid use disorder.
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NewsPCSK9 peptide vaccine lowers LDL cholesterol in preclinical studies
A structure-guided peptide vaccine targeting PCSK9 has demonstrated durable antibody responses and reduced LDL cholesterol levels in mouse models of hypercholesterolaemia, offering a potential long-lasting alternative to current lipid-lowering therapies.
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NewsVitronectin protein identified as key driver of lung scarring
Researchers from UTS and Monash University have identified vitronectin as a key protein driving abnormal lung scarring in idiopathic pulmonary fibrosis, opening a potential new avenue for therapeutic intervention in a disease with very few treatment options.
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NewsPancreas organoids reveal CFTR dysfunction as chronic pancreatitis drug target
Researchers at the Salk Institute have developed a patient-derived organoid platform that identifies distinct biological subtypes of chronic pancreatitis and highlights CFTR as a potential therapeutic target, raising prospects for more personalised treatments.
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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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WebinarBuilding better therapeutic antibodies: the science, progress and problems to solve
As therapeutic antibodies become more complex, where is the field making real progress and which problems are proving hardest to solve? Hear leading experts share their perspectives.
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ArticleOrgan chips move closer to drug discovery pipelines
From uncovering new drug targets to predicting human toxicity, organ chips are showing what they could bring to drug discovery. Professor Donald Ingber of Harvard University discusses where the technology is heading next.
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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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News15-PGDH enzyme inhibition protects brain cells in Parkinson’s disease models
Researchers have identified 15-PGDH enzyme inhibition as a neuroprotective strategy in Parkinson’s disease, with repurposable compounds already in clinical development offering a potential shortcut to disease-modifying therapies.
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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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NewsPeptide vaccine targeting PCSK9 proves effective in cardiovascular disease models
A peptide vaccine targeting PCSK9 has demonstrated sustained antibody responses and reductions in LDL cholesterol and atherosclerotic plaque formation in animal models, offering a potential long-term alternative to current lipid-lowering therapies.
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NewsATRX mutations reprogram chromatin to drive glioma progression
Scientists at MD Anderson Cancer Center have revealed how ATRX mutations restructure chromatin and activate oncogenic developmental pathways in glioma, pointing towards novel therapeutic targets including the HOXA signalling axis.
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NewsMETTL3 protein finds new mechanism driving breast cancer metastasis
Scientists at Umeå University have uncovered a previously unknown function for the RNA-modifying protein METTL3, revealing it plays a distinct role in enabling breast cancer cells to invade surrounding tissue and form metastases – findings that could open new avenues for therapeutic targeting.
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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.


