CRISPR

Shanghai Jiao Tong University is investigating neuroscientist Zilong Qiu after a 6-year-old girl died from a severe immune reaction in an experimental gene-editing trial that went undisclosed.

Jennifer Doudna's team used AI protein design to create synthetic TnpB nucleases that edited human genes more efficiently than the natural enzyme.

The Jennifer Doudna-founded CRISPR biotech filed to go public under ticker SCTX as its lead cholesterol therapy remains early in human testing.

Base editing knockout of NANOG in human embryos shows it is essential for the epiblast, the tissue that forms the body, arriving alongside a separate disease-gene editing study that has renewed debate over heritable genome editing.

The FDA approved expanded use of Vertex's CRISPR therapy Casgevy for patients as young as 2 with sickle cell disease or transfusion-dependent beta thalassemia, making roughly 5,500 more U.S. children eligible.

Lonvoguran ziclumeran met its primary endpoint with an 87% reduction in monthly attacks, the first Phase 3 win for an in vivo CRISPR therapy.

The FDA's June 2 draft guidance lets genome-editing sponsors leverage prior CMC, nonclinical, and clinical knowledge across products, with a 90-day comment window.

Scribe's ELXR platform silences PCSK9 in the liver without cutting DNA; a single low dose sustained LDL reductions for roughly 18 months in primates.

Researchers at the University of Florida and the Hong Kong University of Science and Technology each report a DNA-guided CRISPR-Cas12 platform that targets RNA, reducing off-target effects and enabling attomolar-level pathogen detection.

A University of Utah-led international team reports a new CRISPR nuclease, Cas12a2, that uses RNA guides to trigger widespread DNA shredding and cell death specifically in diseased cells such as KRAS-mutant cancers and HPV-infected cells, while leaving healthy cells unharmed.

First-quarter results from both partners show CASGEVY past 500 treatment starts, a US filing for ages 5 to under 12, and CRISPR Therapeutics' in vivo pipeline advancing across four liver-directed programs.

Intellia Therapeutics will report topline Phase 3 results for lonvoguran ziclumeran in hereditary angioedema on April 27, 2026 — the first Phase 3 readout for any in vivo CRISPR candidate.