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European initiative aims to bring molecular dynamics into mainstream drug discovery
What if the vast amounts of data generated by molecular dynamics simulations could be routinely shared and reused? A new €10 million European initiative aims to do just that, helping researchers gain a deeper understanding of protein behaviour and drug-target interactions.
Pancreas 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.
NTX-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.
New 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.
AUTAC 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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AUTAC 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.
Mechanical forces key to realistic organ-on-chip laboratory models
A new review highlights how physical forces such as blood flow, breathing and tissue stiffness must be replicated alongside biochemical signals to produce physiologically accurate organoid and organ-on-chip models for disease research and drug testing.
New 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.
Gene therapy shows durable correction of methylmalonic acidemia in preclinical study
Preclinical findings from Genespire and SR-TIGET suggest a single administration of a liver-directed lentiviral gene therapy could provide lifelong correction of methylmalonic acidemia, a rare inherited metabolic disorder with no approved disease-targeted treatments.
Stem cell organoids repair heart microvessels in coronary artery disease models
Stanford University researchers have demonstrated that stem cell-derived vascular organoids can regenerate damaged microvessels in the heart, improving cardiac function in a porcine model of ischaemic heart disease and addressing a significant unmet need in coronary artery disease treatment.
Synthetic organiser cells enhance kidney organoid reproducibility and accuracy
Scientists at the University of Southern California have engineered synthetic organiser cells that produce localised Wnt signals to guide kidney organoid development, yielding more reproducible, physiologically accurate structures and revealing a previously unrecognised developmental axis in the human kidney.
KCL-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.
Insilico 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.
Experimental HIV vaccine generates broadly neutralising antibodies in primates
A collaborative research team from LJI, Scripps Research and IAVI has developed an experimental HIV vaccine that successfully generated broadly neutralising antibodies in rhesus macaques, marking a pivotal advance in the 14-year effort to design an effective HIV vaccine.
15-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.
AI 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.
bit.bio launches iPSC-derived cell culture media kits to democratise human cell research
Cambridge-based cell programming company bit.bio has introduced two new media kits for its iPSC-derived ioGlutamatergic Neurons and ioMicroglia, reducing media preparation costs by around 18-fold and simplifying workflows to widen access to human-relevant research models.
XL20 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.
Dual antibody cocktail offers complete Nipah virus protection
Researchers at Mount Sinai have developed the first fully human monoclonal antibody cocktail to provide complete protection against Nipah and Hendra viruses in preclinical models, offering a potential blueprint for treating high-consequence zoonotic pathogens with no approved therapies.
Peptide 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.
ATRX 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.
Fish-inspired sensor detects heartbeat of lab-grown cardiac organoids
A biomechanical well plate inspired by the pressure-sensing lateral line of fish can wirelessly monitor the pulse of multiple lab-grown cardiac organoids simultaneously, offering a scalable new platform for cardiovascular drug testing.
Epilepsy drug sodium valproate could improve vaccine immune responses
A commonly prescribed epilepsy drug has shown striking potential as a vaccine booster in a controlled human trial, more than doubling antibody levels and increasing T cell responses tenfold at a fraction of its standard therapeutic dose.
METTL3 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.
Accessible multiplex imaging workflow advances spatial liver disease research
A new multiplex immunofluorescence workflow using standard laboratory equipment and open-source software enables detailed spatial analysis of liver tissue, organoids and organ-on-a-chip models, lowering barriers to advanced spatial biology in hepatology research.