Six patients with glycogen storage disease type Ia have received a single infusion of BEAM-301, an in vivo base editor ...

Researchers built a three-layer human blood-vessel model that shows how fibroblasts amplify the vascular pathology of ...

Researchers have used CRISPR-Cas9 to remove CD28 from donor T cells and prevent fratricide before equipping them with ...

Wave Life Sciences has reached alignment with the US Food and Drug Administration on a registrational pathway for WVE-006, a GalNAc-conjugated RNA editing oligonucleotide for alpha-1 antitrypsin ...

Researchers in Germany have identified a SOX9-responsive regulatory route to IRF6, a major orofacial-cleft risk gene, in cranial neural crest cells rather than only in oral epithelium. CRISPR-Cas9 ...

The minimal residual disease-positive cohort opens as part of the pivotal-stage development of Soficabtagene Geleucel (Sofi-cel), an off-the-shelf CAR-T cell therapy under evaluation for relapsed or ...

Great to see our founder Jens-Ole Bock speaking at this week's CRISPR Gene Editing Hands-on Workshop... Clinical: Expanded Access Takes SNIPR001 Beyond... CRISPR-Cas base editing altered HTT RNA ...

A gene-edited pig kidney kept a patient with end-stage kidney disease dialysis-free for 271 days, then failed and was removed without leaving the recipient sensitised against human donors. Eighty-two ...

Rationally engineered chimeric attachment sites raise the efficiency with which the serine integrase Bxb1 inserts large DNA cassettes into targeted loci in both human cells and rice. Paired with an ...

Phase 1 study in patients with mCRPC expected to begin enrollment in the fourth quarter of 2026 REDWOOD CITY, Calif.--(BUSINESS WIRE)--Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology ...

Emerging from stealth, Kindred announces peer-reviewed results in The CRISPR Journal, demonstrating the creation of two healthy beagles with a targeted CRISPR-Cas9 edit that eliminates the major dog ...

A six-year-old girl died after receiving an experimental CRISPR-Cas base-editing treatment for a nonfatal neurodevelopmental disorder in China. The fatal immune reaction followed preclinical safety ...