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Gene editing outcomes set up FDA submissions in Duchenne
Regenxbio said its Duchenne muscular dystrophy (DMD) gene therapy reached the necessary bar in a pivotal study, with the company aiming for an FDA submission in 2027. The milestone centers on...
AAV safety signal triggers renewed focus on genome integration
Researchers at Children’s Hospital of Philadelphia (CHOP) described a rare brain-tumor case linked to adeno-associated virus (AAV) integration after intracisternal magna vector delivery. The work,...
AI-enabled drug design gets its biggest venture infusion yet
Isomorphic Labs closed a $2.1 billion Series B to scale its AI-driven small-molecule drug design engine, IsoDDE. The funding makes the round one of the largest ever for a biotech startup and...
FDA clears new solid-tumor genomic profiling test
Datar Cancer Genetics received FDA clearance for CellDx-Tissue, a comprehensive genomic profiling assay for solid tumors. The dual-analyte test profiles targeted DNA and RNA alterations using a...
Regulatory milestone: BeOne’s BCL2 drug wins U.S. accelerated approval
BeOne Medicines’ sonrotoclax (branded as Beqalzi) received U.S. FDA accelerated approval for relapsed or refractory mantle cell lymphoma (MCL) after treatment with a BTK inhibitor. The approval...
Clinical hold and program reprioritization in rare diseases
Alumis said it is pulling back from a would-be rival to Amgen’s Tepezza after assessing prospects for the asset it acquired. The company chose to punt the phase 3 path that had been envisaged...
Venture deals in in vivo and predictive platforms
Veristat launched InStat, an automated biostatistics platform designed to cut clinical trial readout timelines from weeks to days. The CRO said the system produces submission-ready tables,...
Bioscience M&A continues beyond large pharma
Biopharma M&A is showing a broader buyer base, with mid-size American drugmakers and family-owned or foundation-governed European players stepping up deal hunting. The coverage points to a market...
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...
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...
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...
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...
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...
In vivo CAR-T funding boost for next wave of cell therapies
Create Medicines raised $122 million to expand its in vivo CAR-T platform, aiming to advance programs across cancers and autoimmune disease. The Series B funding co-led by Arch Venture Partners,...
Biotech financing megadeal for AI drug discovery model
Isomorphic Labs secured a $2.1 billion Series B round to scale its AI predictive drug discovery engine and advance its internal pipeline toward the clinic. Reports describe a syndicate led by...
FDA approval: BeOne’s Beqalzi wins accelerated nod in mantle cell lymphoma
BeOne Medicines received US accelerated approval for sonrotoclax (Beqalzi), a next-generation BCL2 inhibitor targeting relapsed or refractory mantle cell lymphoma following BTK inhibitor therapy....
Massive pharma-developer deal: BMS and Hengrui expand oncology and immunology collaboration
Bristol Myers Squibb and Hengrui Pharmaceuticals signed a collaboration that covers 13 early-stage programs spanning oncology/hematology and immunology, with potential deal value exceeding $15...
AI-enabled trial operations: Veristat launches automated biostatistics platform
Veristat launched InStat, a fully automated biostatistics platform aimed at compressing clinical trial data readout timelines from weeks to days. The software is designed to deliver...
COVID-19 and infectious disease surveillance
Researchers unveiled a wastewater surveillance approach that tracks SARS-CoV-2 genetic diversity rather than relying on viral abundance alone, aiming to improve early outbreak forecasting. The...
Biotech funding and platform scale-up
Isomorphic Labs announced a $2.1 billion Series B financing led by Thrive Capital, describing it as a step-change in scaling its AI drug design engine, IsoDDE. The company framed the round as a...