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Whole-transcriptome diagnostics validated: 1,233 FFPE tumor cohort reported
Researchers led by Ball, Beck, and Wlochowitz published a large‑scale diagnostic study in the British Journal of Cancer showing that whole‑transcriptome sequencing performs robustly across 1,233...
Sarepta sales miss opens fresh gene‑therapy safety questions – stock tumbles
Sarepta Therapeutics reported preliminary Q4 2025 product sales that fell short of analyst expectations, driven by weaker-than-forecast revenue for its AAV gene therapy Elevidys and a decline in...
TGFβ drives shape switch in dormant lung metastases… immune evasion mechanism revealed
Researchers at Memorial Sloan Kettering Cancer Center reported that TGFβ signaling induces an atypical epithelial‑to‑mesenchymal transition that ultimately converts disseminated lung...
Enterococcus faecalis redox metabolism implicated in chronic wound failure – new therapeutic target
An international team led by NTU Singapore and the University of Geneva identified extracellular electron transport (EET) in Enterococcus faecalis as a mechanism that generates reactive oxygen...
Fratricide‑driven, unedited CD7 CAR‑T shows efficacy in T‑cell leukemia models
A team publishing in the Journal of Translational Medicine described fratricide‑driven, unedited CD7 CAR‑T cells as a promising therapy for T‑cell leukemia. The approach leverages the tendency of...
Two‑step genome editing creates full‑length humanized mouse models – new tool for in vivo human gene studies
Researchers developed a two‑step genome‑editing workflow to replace mouse loci with full‑length human sequences, enabling creation of humanized mouse models that preserve human regulatory...
Real‑time metabolic monitoring enters cell and gene therapy manufacturing workflows
PHCNA coverage highlights the deployment of real‑time cell metabolic monitoring technologies in cell and gene therapy (CGT) manufacturing to enable earlier interventions and tighter process...
FDA signals support for Hemostemix basket protocol approach
Regulatory commentary and a community submission note that Hemostemix received favorable engagement from the FDA for its basket protocol strategy, according to a public post. The interaction...
Legal questions surface over FDA’s new expedited drug pathway – sign‑off and oversight in focus
Reporting flagged legal and procedural concerns about the FDA’s new expedited drug program, including uncertainty about who must sign off on approvals and whether standard review steps can be...
Advocates push Congress to preserve NIH FY2026 funding at $48.7B – urgent call to action
Advocacy groups are urging House representatives to support a FY2026 appropriations bill that maintains NIH funding at the Senate‑approved $48.7B and preserves language preventing caps on indirect...
AI‑trained antibody probes let scientists see protein activity inside living cells
Colorado State University researchers used artificial intelligence to design modified antibodies that fluoresce inside living cells, enabling real‑time visualization of intracellular protein...
FDA backs Hemostemix: regulator signals support for basket protocol
Hemostemix received formal support from the U.S. Food and Drug Administration for its basket protocol approach, the company announced. The FDA’s endorsement clears a regulatory hurdle for testing...
Sarepta’s Elevidys sales fall short – shares slide amid gene therapy safety cloud
Sarepta Therapeutics disclosed preliminary Q4 2025 results showing Elevidys net product revenue of $110.4 million, down 17% from Q3 and missing analyst consensus, prompting an immediate share...
TGFβ-induced shape shift: dormant lung cancer cells evade immune attack
Memorial Sloan Kettering researchers reported in Nature Cancer that disseminated lung adenocarcinoma cells use a TGFβ-driven program to enter dormancy and adopt a rounded morphology that reduces...
Enterococcus faecalis weaponizes redox metabolism: EET activation stalls wound healing
An international team led by NTU Singapore and the University of Geneva published in Science Advances that Enterococcus faecalis impairs wound repair by generating reactive oxygen species via...
Fratricide-driven CD7 CAR‑T: unedited cells demonstrate efficacy in T‑cell leukemia
Researchers publishing in the Journal of Translational Medicine described a fratricide-driven approach using unedited CD7 CAR‑T cells to treat T‑cell leukemia. The strategy leverages intrinsic...
Real-time metabolic monitoring: firms deploy sensors to stabilize cell and gene therapy manufacture
Industry practitioners highlighted by PHCNA emphasize that real-time metabolic monitoring is moving from pilot to production in cell and gene therapy (CGT) workflows. Tia Harmon noted technologies...
Two-step genome editing enables full-length humanized mouse models
Researchers reported a two-step genome editing strategy that creates full-length humanized mouse models, overcoming limitations in regulatory sequence divergence between species. The method...
AI-tuned antibodies act as intracellular lights: researchers accelerate in-cell probes
Colorado State University researchers used artificial intelligence to modify antibodies so they function as high-signal probes inside living cells, enabling dynamic readouts of gene-expression...
Tetraspanin immunocapture: high‑depth proteomics of CSF extracellular vesicles
Scientists described a tetraspanin-based immunocapture method that substantially increases proteomic depth for extracellular vesicles (EVs) isolated from cerebrospinal fluid (CSF). The technique...