Shinshu University Hospital's expertise in CAR T-cell therapy R&D and clinical application will be combined with Teijin Regenet's manufacturing and quality-management expertise cultivated through its CDMO...
Manufacturing hurdles are just one challenge the industry must overcome to commercialize personalized mRNA cancer vaccines.
Encoded Therapeutics’ lead asset ETX101 is a gene therapy being tested for Dravet syndrome. Phase 1/2 data showed substantial reductions in seizures.
Before the end of September, UniQure is expected to report four-year results from the closely tracked clinical trial of its gene therapy for Huntington’s disease.
Nature Biotechnology, Published online: 10 September 2026; doi:10.1038/s41587-026-03289-9Recent patents relating to systems, methods and compositions for use in synthetic biology.
BIOCENTURY | EMERGING COMPANY PROFILEBRAINCHILD: BRINGING CAR TS DIRECTLY TO THE BRAINRepeat dosing into the CSF is designed to sidestep the toxicities that have limited CNS-directed...
Intravenous gene therapy extended lifespan, reduced neurological symptoms, and improved disease markers in a Sandhoff disease cat model, supporting further investigation of systemic AAV gene therapy...
BMS' arlo-cel has shown activity in a trial that could underpin filings in multiple myeloma settings where the four main drug classes have failed.
Bristol Myers Squibb is positioning arlo-cel for quadruple-class exposed patients, which could help reduce competition with an upcoming multiple myeloma CAR T therapy from Gilead Sciences,...
Nature Reviews Drug Discovery, Published online: 09 September 2026; doi:10.1038/d41573-026-00125-2To unlock the ‘plug-and-play’ potential of extrahepatic delivery of oligonucleotide-based drugs, the field must bridge the gap...
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