All Small Molecules articles
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ArticleTargeting childhood myopia progression with 7-methylxanthine
Childhood myopia researcher Dr Klaus Trier explores how 7-methylxanthine targets scleral remodelling and axial elongation, and its potential to slow disease progression and reduce the risk of high myopia.
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NewsAGR2 and FOXM1 identified as prognostic and therapeutic targets in NF-PanNETs
A new study using single-cell RNA sequencing has identified AGR2 as a potential prognostic marker and FOXM1 as a druggable target in non-functional pancreatic neuroendocrine tumours, offering new tools to stratify patients and guide treatment strategies.
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ArticleWhy intracellular targets remain a major drug discovery challenge – and how they’re becoming druggable
Many biologically important intracellular targets remain difficult to drug. Dr Rab Prinjha examines how screening within living cells could help tackle them.
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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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NewsInsilico Medicine nominates AI-designed candidate for ocular diseases
Insilico Medicine has nominated ISM9077, an AI-designed small molecule targeting pathological inflammation, as its 32nd preclinical candidate since 2021, with potential applications across dry AMD, uveitis and dry eye disease.
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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.