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Duchenne gene therapy pathway toward FDA submission
Regenxbio said its Duchenne muscular dystrophy gene therapy RGX-202 met the bar in a pivotal trial, producing sufficiently high levels of a miniaturized muscle protein and paving the way for an...
In vivo CAR-T platform financing expansion
CREATE Medicines raised a $122 million Series B to advance its in vivo CAR-T platform across autoimmune disease and oncology, pushing programs including CRT-402 (CD19 CAR T for autoimmune disease)...
Big Pharma partnership with Hengrui covering 13 programs
Bristol Myers Squibb and Hengrui Pharma signed a collaboration and license agreement covering 13 early-stage programs spanning oncology/hematology and immunology, valued at up to $15.2 billion....
Company exit strategy in oncology partnership after takeout delay
Aptose Biosciences ended its blood cancer collaboration tied to CG-806 after its pending takeover by Hanmi Pharmaceutical constrained further development options. The deal unwound after Aptose...
FDA clearance for solid tumor genomic profiling diagnostic
Datar Cancer Genetics received FDA clearance for its CellDx-Tissue comprehensive genomic profiling test for solid tumors. The dual-analyte assay combines targeted next-generation sequencing of DNA...
Cell and gene therapy marketplace for shelved programs
ASGCT and the Orphan Therapeutics Accelerator launched CGTxchange, an AI-enhanced clearinghouse intended to reactivate shelved clinical-stage cell and gene therapy programs. The platform is...
Expansion of in vivo adaptive analytics for clinical trial readouts
Veristat rolled out InStat, a fully automated biostatistics platform positioned to compress clinical trial data readouts from weeks to days. The company said sponsors can receive submission-ready...
Large Series B for AI drug design scaling
Isomorphic Labs, the Google-backed AI drug design company, secured a $2.1 billion Series B round led by Thrive Capital, a scale-up signaling intensified competition in AI-driven small-molecule...
Clinical holds and program execution risk
Aardvark Therapeutics said it is preparing to unblind data from its Prader-Willi syndrome (PWS) phase 3 program after the FDA imposed a full clinical hold tied to a cardiac safety signal in...
In vivo CAR-T funding accelerates autoimmune and oncology pipelines
CREATE Medicines secured a $122 million Series B to advance its in vivo immune programming platform across autoimmune disease and oncology. The round was co-led by existing investors Arch Venture...
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...