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VGLUT3 gene therapy restores hearing in aged mice
Researchers delivered a VGLUT3 gene therapy to inner hair cells and restored auditory function in aging mice, demonstrating a potential route to treat age‑related hearing loss by repairing...
Scientists immortalize bovine cells without genetic engineering — cultivated beef scale‑up
A team at Hebrew University and Believer Meats reported a non‑genetically modified method to extend bovine cell self‑renewal by activating telomerase and PGC1α, producing stable, self‑renewing cow...
Machine learning predicts DCD donor death — slashes futile liver procurements
Stanford Medicine and Kyoto University researchers developed a machine‑learning model that predicts whether donation‑after‑circulatory‑death (DCD) donors will die within the time window suitable...
Merck stakes $9.2B on long‑acting flu drug — Cidara takeover
Merck announced a $9.2 billion agreement to acquire Cidara Therapeutics to secure CD388, a long‑acting, broadly active influenza prevention candidate now in a Phase III trial. Merck said it will...
FDA tightens Elevidys label: boxed warning, use narrowed
The U.S. Food and Drug Administration revised the label for Sarepta’s Duchenne gene therapy Elevidys, adding a boxed safety warning and restricting use to ambulatory patients. The agency also...
AAV arms race... point‑of‑care antibody test and new capsids emerge
Two developments targeted at adeno‑associated virus (AAV) gene therapy delivery surfaced: a point‑of‑care test for anti‑AAV8 binding antibodies and discovery of AAV variants that markedly improve...
Intranasal nanogel vaccine slows HPV‑driven cervical tumors — preclinical win
Researchers at Chiba University reported a cationic nanogel nasal therapeutic vaccine (cCHP‑E7 + c‑di‑AMP) that induces localized T‑cell responses and limits HPV16 E7‑driven tumor growth in mice...
Prime‑editing inversion method scales chromosomal rearrangements — PIE reported
Scientists published Prime‑Editing‑Based Inversion with Enhanced Performance (PIE), a technique that enables efficient, large‑scale inversion of genomic DNA at chromosomal scale in mammalian...
Biomimetic mRNA delivery restores PTEN — targeted immunotherapy for colorectal cancer
Researchers developed a biomimetic mRNA delivery system that restores PTEN expression in colorectal tumors and enhances targeted immunotherapy activity. The platform delivers PTEN mRNA to tumor...
AI flags resistance — CarbaDetector spots carbapenemase from routine tests
A machine‑learning model named CarbaDetector was reported to detect carbapenemase‑producing Enterobacterales (CPE) directly from disk diffusion antibiotic susceptibility tests. The algorithm...
Next‑gen blood thinner milvexian fails key trial — anticoagulation program hit
Bristol Myers Squibb and Johnson & Johnson announced a Phase III futility readout in their acute coronary syndrome trial of milvexian, a selective Factor XIa inhibitor, and will halt that study....
Medicxi raises €500M fund — backers target creation‑stage biotech
European venture firm Medicxi closed a €500 million Fund V focused on company creation and selective later‑stage investments, according to the firm. The vehicle targets product‑focused startups...
AMP issues draft guidelines on cancer sequencing variant interpretation
The Association for Molecular Pathology (AMP) released draft guidelines for interpreting and reporting cancer sequencing variants, covering biomarkers that predict therapy response or resistance,...
Merck snaps up Cidara for $9.2B — late-stage flu antiviral in hand
Merck agreed to acquire Cidara Therapeutics for about $9.2 billion to secure CD388, a long-acting antiviral designed to prevent seasonal and pandemic influenza. CD388 — a small-molecule...
FDA narrows Elevidys label — boxed warning, ambulatory restriction added
The FDA revised the prescribing information for Sarepta’s Duchenne muscular dystrophy gene therapy Elevidys, adding a boxed safety warning and restricting use to ambulatory patients. The agency...
Factor XIa program stumbles — next‑gen blood thinner fails Phase 3 readout
Bristol Myers Squibb and Johnson & Johnson halted a Phase III trial of their factor XIa inhibitor milvexian after an interim analysis showed the study was unlikely to meet its primary endpoint in...
New AAV variants boost vascular gene delivery — vascular transduction improved
Researchers led by Maria Stamataki reported discovery of engineered adeno‑associated virus (AAV) variants that markedly improve transduction of human vascular endothelial cells. The work,...
Prime‑editing inversion (PIE) enables chromosome‑scale DNA rearrangements
A new genome engineering method, Prime‑Editing‑Based Inversion with Enhanced Performance (PIE), allows researchers to induce large chromosomal inversions in mammalian cells with improved...
Intranasal nanogel therapeutic vaccine slows HPV‑driven cervical cancer in animals
Chiba University researchers published preclinical data in Science Translational Medicine on a cationic nanogel‑based intranasal vaccine targeting HPV16 E7 oncoprotein. The cCHP‑E7 + c‑di‑AMP...
Terasaki, Caltech land $2.8M CIRM grant to model early human embryo formation
The Terasaki Institute for Biomedical Innovation and Caltech secured a $2.8 million Discovery Stage Research grant from CIRM to advance studies of early human embryo formation. The funded project...