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First-in-class drug targets ‘undruggable’ MYC in blood cancers
A first-in-class experimental drug has demonstrated preclinical activity against treatment-resistant blood cancers by exploiting a newly identified vulnerability between MYC and GSPT1, offering a potential route to one of oncology’s most elusive targets.
IL-17 identified as potential immunotherapy biomarker in ovarian cancer
A new study has identified IL-17 as a potential biomarker for immunotherapy response in a small subset of clear cell ovarian cancer cases, offering a possible route to personalised treatment for a disease historically resistant to immune-based therapies.
Exhausted T cells escape tumours to drive lasting cancer immunity
A preclinical study has identified a subset of exhausted, LAG3-expressing T cells capable of migrating out of tumours to establish long-term immune memory, with potential implications for LAG3-targeting cancer immunotherapies.
NUS researchers identify molecular switch driving TNBC treatment resistance
Researchers at the National University of Singapore have identified DP103 as a potential molecular master switch sustaining tumour growth, cancer stem cells and treatment resistance in triple-negative breast cancer, with findings suggesting it could serve as a predictive biomarker for the investigational oral therapy RX-5902.
First multi-responsive foldamer-based gel offers new approach to controlled drug delivery
Researchers at the University of Birmingham have engineered a switchable gel built from synthetic foldamer molecules that responds to light, heat and acid — a platform with potential implications for controlled drug delivery and targeted therapeutic release.
Patient-specific brain tumour chip models vascular drug barriers
A microfluidic chip combining patient-derived glioblastoma cells with their tumour vascular environment could give drug developers a more realistic preclinical model for predicting treatment response – and may help explain why genetically similar patients respond differently to the same therapy.
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First multi-responsive foldamer-based gel offers new approach to controlled drug delivery
Researchers at the University of Birmingham have engineered a switchable gel built from synthetic foldamer molecules that responds to light, heat and acid — a platform with potential implications for controlled drug delivery and targeted therapeutic release.
Patient-specific brain tumour chip models vascular drug barriers
A microfluidic chip combining patient-derived glioblastoma cells with their tumour vascular environment could give drug developers a more realistic preclinical model for predicting treatment response – and may help explain why genetically similar patients respond differently to the same therapy.
AGR2 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.
Multi-target screening refines ADC development for cervical cancer
A large-scale transcriptomic study has pinpointed distinct cell-surface targets for antibody-drug conjugate development across cervical cancer subtypes, revealing how tissue-of-origin comparisons and subpopulation analysis can uncover targets missed by conventional screening approaches.
Human heart organoids with functioning valves model cardiac valve disorders
Researchers have engineered human heart organoids complete with functioning valves, creating a physiologically relevant platform to study valve disorders including mitral valve prolapse and valve calcification, and potentially accelerating the search for new treatments.
Automated MRI pipeline standardises preclinical stroke damage measurement
An automated imaging pipeline developed at USC’s Stevens Neuroimaging and Informatics Institute can measure stroke-related brain tissue damage from MRI scans with accuracy matching human experts, offering a scalable, standardised tool for preclinical drug evaluation across multi-site research networks.
Scientists identify a new vaccine design strategy for stronger HIV immunity
Two preclinical studies published in Science Translational Medicine suggest that the binding strength and structural repetitiveness of vaccine nanoparticles could be a critical design parameter in the long-running effort to generate broadly neutralising antibodies against HIV.
Meis2 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.
Nanomedicine for glioblastoma: overcoming the blood-brain barrier
A new review published in Chinese Neurosurgical Journal examines how nanoscale drug delivery systems could help overcome one of the most persistent obstacles in glioblastoma treatment – getting therapeutics across the blood–brain barrier and into tumour tissue.
Tissue chips target immunotherapy toxicity prediction without animal models
Researchers at the University of Rochester have developed human cell-based tissue chips designed to predict serious immunotherapy toxicities, including cytokine release syndrome, and have secured entry into the FDA’s ISTAND pilot programme.
How CAR T cells are being engineered to target tumour’s ‘soft spot’
A preclinical study published in Nature Biomedical Engineering describes a synthetic biology approach that converts the physical softness of cancer cells into a targetable signal for CAR-T immunotherapy, potentially addressing a key resistance mechanism in solid tumours.
AI 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.
Insilico 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.
AI designs functional bacteriophage genomes to overcome bacterial resistance
Scientists have used artificial intelligence to design complete, functional bacteriophage genomes from scratch, with engineered phages capable of overcoming resistance in bacteria that had defeated naturally occurring viruses — marking a significant step towards AI-driven whole-genome engineering.
AI 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.
New 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.
What Google’s AI talent exodus could mean for future drug discovery
The departure of leading AI researchers from Google to found Discovery Loop raises important questions about the future direction of artificial intelligence in scientific research and drug development.
Targeting 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.
What Oblenio’s Phase I trial means for autoimmune drug discovery
Oblenio Bio has dosed the first patients in a Phase 1a trial of LBL-051, a tri-specific T cell engager designed to simultaneously target CD19, BCMA and CD3 and eliminate both B cells and plasma cells in a bid to achieve durable immune reset in refractory autoimmune diseases.
VY1706 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.