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What’s in Today’s Brief? (May 15th Preview)
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CRISPR tech shift for RNA editing
University of Florida researchers reported a new CRISPR approach in Nature Biotechnology that uses DNA—not RNA—to guide Cas12 nucleases to cellular RNA targets. The platform, called ΨDNA, uses a DNA-based guide scaffold engineered to mimic the crRNA architecture, enabling Cas12a and Cas12i1 to bind RNA and trigger activity via single-stranded DNA trans-cleavage. The team framed the work as a conceptual shift in controlling transcriptomes without the instability challenges seen in RNA-guided systems. In clinical-sample testing, the system achieved 100% accurate hepatitis C virus RNA detection (as reported by the authors). In human cell line experiments, ΨDNA delivered 70–95% knockdown of endogenous transcripts, with mechanistic drivers including ribosome stalling and RNase H1 recruitment.
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Regulatory flashpoint: FDA hold for PWS gene target taste agonist program
Aardvark Therapeutics said the FDA imposed a full clinical hold on its Prader-Willi syndrome program, pausing trials across ARD-101 and related studies following a cardiac safety signal in healthy volunteers. The company had previously paused work voluntarily after QRS prolongation thresholds were observed at the phase 3 dose without titration, and later expanded the pause to ARD-201. With the hold now in place, Aardvark plans to unblind data from its phase 3 ARD-101 study and associated open-label extension. The company said the move is intended to assess the “totality of available efficacy and safety data” to support next-step decisions and preserve options for a financing or partnership window.
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Alzheimer’s tau targeting resumes despite phase 2 endpoint miss
Biogen decided to advance diranersen (BIIB-080) into registrational development for early Alzheimer’s disease even after missing a phase 2 primary endpoint. In the Celia trial, low-dose diranersen outperformed higher doses on cognition measures while the primary dose-response goal on Clinical Dementia Rating–Sum of Boxes at week 76 was not met. Biogen reported reductions in cerebrospinal fluid tau and tau pathology alongside cognitive slowing signals, and the company characterized biomarker and efficacy data as supportive of moving into phase 3. Analysts cited uncertainty around the dose-response pattern, underscoring the need for clearer regimens in late-stage studies.
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FDA clears solid-tumor comprehensive genomic profiling test
Datar Cancer Genetics announced FDA clearance for its CellDx-Tissue comprehensive genomic profiling assay to profile DNA and RNA from solid tumors. The dual-analyte targeted next-generation sequencing test assesses single nucleotide variants, small insertions/deletions, and ERBB2 gene amplification across 517 cancer-associated genes, with RNA-based calls for ALK, RET, and ROS1 fusions. The company said the test can be run on formalin-fixed, paraffin-embedded samples and is performed globally in CAP- and CLIA-accredited laboratories. Datar positioned the clearance as a bridge between genomic complexity and clinical decision-making as targeted therapies expand.
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Cancer diagnostics reimbursement win: Medicare expands Personalis NeXT Personal MRD coverage
Personalis shares rose after CMS expanded Medicare coverage for the company’s NeXT Personal minimal residual disease assay to include immunotherapy monitoring across late-stage solid tumors. The decision was described as supported by evidence from a study with Spain’s Vall d’Hebron Institute of Oncology showing sufficient sensitivity for ongoing surveillance and outcome prediction during immunotherapy. The assay had already gained Medicare coverage for recurrence monitoring in stage II–III breast cancer and stage I–III non-small cell lung cancer. Personalis said IO reimbursement could create additional upside beyond current guidance.
...and 5 more selected Biotech stories in today’s full edition — or archive.
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