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Slowing intracellular transport boosts potential for RNA therapies
Researchers at the University of Basel and Roche have discovered that slowing the intracellular transport of RNA-based drugs significantly improves their therapeutic impact.
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Fixing drug discovery’s most persistent problem with AI
AI will not replace drug discovery, but it might finally fix one of its most frustrating bottlenecks. Read how a targeted approach to ADMET is cutting through the noise.
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New mouse model mimics rare GRIN2D disorder
Researchers at Tel Aviv University have developed a new mouse model that accurately mimics a rare and severe genetic disorder caused by mutations in the GRIN2D gene – allowing for study of the disease’s progression and the testing of potential drug therapies.
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LINC01235 identified as driver of aggressive breast cancer
Researchers at Cold Spring Harbor Laboratory have demonstrated that an obscure RNA molecule, LINC01235, plays a crucial role in the progression of aggressive triple-negative breast cancer (TNBC) – potentially allowing for the development of targeted therapies against it.
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Leukaemia-on-a-chip mimics bone marrow for better CAR T testing
A new “leukaemia-on-a-chip” device replicates human bone marrow and immune interactions, enabling researchers to observe CAR T cell therapies in action - potentially allowing for more personalised treatment strategies for leukaemia patients.
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QTX153 reverses Rett symptoms and crosses key drug delivery barrier
QTX153, a novel HDAC6 inhibitor, has shown significant symptom reversal in preclinical models of Rett syndrome. This represents progress toward a therapy for a condition with no approved options.
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Small molecule therapy improves islet transplant success in diabetes
A study from Weill Cornell Medicine shows that pre-treating pancreatic islet cells with a small molecule cocktail significantly improves survival after transplantation in type 1 diabetes models. The approach could help make donor cells go further and transplants more efficient.
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Virtual cell model rankings just got a major upgrade
Shift Bioscience has published a new study introducing enhanced metrics and baselines for evaluating virtual cell models - boosting gene target discovery and accelerating its rejuvenation therapeutics pipeline.
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New data backs Myo4 for obesity treatment
MitoRx Therapeutics has announced new preclinical data for its small molecule Myo4, showing restored insulin sensitivity and enhanced fat loss with muscle preservation in an obesity model - offering a potential alternative to GLP-1-based therapies.
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Liver organoids grow functional blood vessels in lab breakthrough
Scientists have successfully grown liver organoids with fully functional blood vessels- potentially allowing for the development of new treatments for haemophilia and liver disease.
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SOD1 protein found to trigger treatable Parkinson’s progression
Scientists at the University of Sydney have discovered a malfunctioning brain protein linked to Parkinson’s - which could lead to new therapies for the debilitating condition in the future.
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Meet the hemifusome: a new organelle with big impact
Scientists have discovered a previously unknown organelle inside human cells - the hemifusome - that could change our understanding of rare genetic disorders.
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Disabling the SETD1B enzyme halts leukaemia cell growth
Japanese researchers have identified the epigenetic enzyme SETD1B as a key driver of aggressive acute myeloid leukaemia (AML) – which could lead to new treatment strategies targeting the cancer’s underlying biology in the future.
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How bowel cancer beats treatment – and how AI can stop it
Scientists have developed a new AI-guided tool that predicts how bowel cancer becomes resistant to treatment – which could lead to development of new personalised therapies.
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Protein folding milestone achieved with quantum tech
Kipu Quantum and IonQ have set a new benchmark in quantum computing by solving the most complex protein folding problem ever tackled on quantum hardware – creating potential for real-world applications in drug discovery.
