All Drug Targets articles
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NewsMeis2 gene identified as potential therapeutic target for neurodevelopmental disorders
A gene therapy targeting parvalbumin inhibitory neurons has restored experience-dependent plasticity and reduced seizure frequency in a mouse model of neurodevelopmental disorders, offering a potential new avenue for treating autism spectrum disorder and epilepsy.
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ArticleFibroblast barrier linked to immunotherapy resistance in colorectal cancer
Why do some colorectal cancers resist immunotherapy? Analysis of patient tumour samples has identified a population of fibroblasts that could help explain treatment resistance.
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NewsAI identifies experimental drug targeting undruggable cancer protein
Mayo Clinic researchers have used AI to screen nearly 40,000 compounds and identify a small molecule inhibitor targeting GIPC1, a protein previously considered undruggable, with preclinical results showing slowed tumour growth and enhanced chemotherapy response.
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ArticleWhy single-cell foundation models have underdelivered and what drug discovery needs instead
Foundation models have delivered breakthroughs in protein biology, but single-cell models have struggled to match them. What is holding them back?
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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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NewsVY1706 Alzheimer’s gene therapy achieves 75 percent tau reduction in preclinical study
Voyager Therapeutics has presented six-month GLP toxicology data for VY1706, its investigational tau-targeting gene therapy, showing durable tau reductions of up to 75 percent and a clean safety profile in non-human primates, as the company prepares to begin first-in-human dosing in the second half of 2026.
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NewsMitochondrial microprotein discovery reveals new Type 2 diabetes genetic link
A newly identified mitochondrial microprotein, MENTSH, has been linked to increased Type 2 diabetes susceptibility in Mexican and Mexican American populations, pointing towards a potential precision medicine target.
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NewsBMP3 protein identified as therapeutic target for pulmonary arterial hypertension
Scientists at the Fralin Biomedical Research Institute have identified bone morphogenetic protein 3 as a protective factor in lung blood vessels, with preclinical data showing that restoring its levels can reverse hallmark features of pulmonary arterial hypertension.
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ArticleInside one of the most comprehensive human proteome atlases to date
Understanding where proteins are expressed throughout the body is critical for selecting better drug targets. Here’s how a new human proteome atlas could improve target selection, predict toxicity and support drug repurposing.
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NewsHow dendritic cells organise tumour immunity – and why drug developers should take notice
A new study published in Science identifies a specialised dendritic cell subset as the cellular architects behind tertiary lymphoid structures, offering a potential new target for boosting anti-tumour immunity and converting immunologically cold tumours into treatment-responsive ones.
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NewsLandmark fibromyalgia study highlights neurological targets for new therapies
A landmark genome-wide study of more than 2.5 million adults has identified 26 genomic regions linked to fibromyalgia, confirming a neurological basis for the condition and opening new avenues for targeted drug discovery.
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WebinarOligonucleotide therapeutics safety considerations across drug development
As oligonucleotide therapeutics continue to advance, their safety assessment presents challenges that differ from traditional drug modalities. Discover how nonclinical safety studies, evolving regulatory expectations and real-world case examples can help you navigate these challenges with greater confidence.
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NewsAmino acid sensor discovery could unlock new targeted cancer therapies
Researchers have uncovered how cells sense amino acids and activate the mTORC1 growth pathway, identifying LARS1 phosphorylation as a critical upstream switch with implications for more precise cancer drug development.
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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.


