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Glymphatic gene therapy platform targets brain’s glial cells precisely
A new gene therapy platform exploiting the brain’s glymphatic transport system and engineered AAV5 vectors could offer a targeted treatment route for multiple sclerosis, Huntington’s disease and rare white matter disorders.
Triple 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.
Mitochondrial 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.
BMP3 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.
Insilico Medicine launches AI drug discovery benchmarking platform
Insilico Medicine has unveiled a benchmarking platform designed to assess whether AI models can perform genuine drug discovery tasks, using decontaminated real-world datasets and proprietary validated programmes to move beyond inflated benchmark scores.
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Insilico Medicine launches AI drug discovery benchmarking platform
Insilico Medicine has unveiled a benchmarking platform designed to assess whether AI models can perform genuine drug discovery tasks, using decontaminated real-world datasets and proprietary validated programmes to move beyond inflated benchmark scores.
Fullscope-seq maps transcript isoforms with spatial single-cell resolution
Researchers have developed Fullscope-seq, a long-read spatial transcriptomics platform that maps alternative transcript isoforms within intact tissues at single-cell resolution – opening new possibilities for target identification and precision biomarker discovery.
Researchers develop AI system to accelerate tuberculosis drug discovery
Researchers at Texas A&M have developed AI-driven platforms to help scientists navigate the bottlenecks of tuberculosis drug discovery, from eliminating nuisance compounds to unlocking years of archived research data.
What regenerative medicine can learn from multifunctional biomaterials
Researchers at the Indian Institute of Technology Gandhinagar have developed an injectable hydrogel incorporating a novel cerium–rutin metal–phenolic nanocomplex that simultaneously targets oxidative stress, inflammation and bacterial contamination in deep wounds—without the use of antibiotics.
Blood test for protein misfolding identifies Alzheimer’s risk years before symptoms
A blood-based biomarker measuring amyloid beta protein misfolding has demonstrated superior predictive accuracy for pre-symptomatic Alzheimer’s disease compared with p-tau 217, achieving an AUC of 0.87 when combined with genetic and demographic data.
How 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.
Landmark 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.
Doxycycline found to disrupt bacterial ribosomes via two unknown mechanisms
Researchers have identified two previously unknown mechanisms by which doxycycline inhibits bacterial protein synthesis, findings that could inform the design of next-generation antibiotics against drug-resistant pathogens.
Non-endocytic delivery bypasses endosomal barriers for gene therapy
A new review examines how physical, bioengineered and biomimetic delivery platforms that bypass conventional endocytic uptake pathways could transform the clinical development of gene therapies, mRNA treatments and protein-based medicines.
Amino 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.
Oral 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.
3D 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.
Insilico 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.
ELRIG appoints two industry veterans to their Scientific Advisory Board
Not-for-profit drug discovery organisation ELRIG has appointed Dr Hitesh Sanganee and Dr Marcus Schindler to its Scientific Advisory Board, bringing decades of combined R&D, venture investment and pharmaceutical leadership expertise to help shape the organisation’s future scientific programmes.
New 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.
New bispecific T cell engager platform targets ovarian cancer in preclinical studies
Scientists at The Wistar Institute have engineered a DNA-delivered bispecific T cell engager using a ‘knob-into-hole’ antibody platform, demonstrating improved persistence and dual-antigen targeting in preclinical ovarian cancer models.
Tumour organoids may expand access to targeted cancer drugs
Two studies from Weill Cornell Medicine demonstrate that patient-derived tumour organoids can accurately replicate cancers long-term and incorporate immune microenvironments, offering new potential for personalised treatment selection.
Spatial map reveals hidden complexity of bladder cancer tumours
Researchers at MD Anderson Cancer Center have used spatial transcriptomics to chart the internal architecture of muscle-invasive bladder cancer, revealing that luminal and basal cancer cell programmes coexist within single tumours – a finding with significant implications for treatment stratification.
Crown Bioscience joins C-Path coalition advancing regulatory NAMs acceptance
Crown Bioscience has joined the Critical Path Institute’s New Approach Methodologies Developer Coalition, bringing patient-derived model expertise and biomarker capabilities to efforts aimed at establishing regulatory qualification frameworks for human-relevant research tools.
New 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.