The first approved CRISPR medicine has now shown similarly strong results in children as young as five years old with sickle cell disease and transfusion-dependent β-thalassaemia. In a pair of phase 3 ...
Screening ionisable lipids with a gene-editor-sized RNA identified LC-1, an LNP component that retained potency as cargo ...
Incyte will share updated clinical single-cell data characterizing its calreticulin-targeted therapy for myeloproliferative ...
Researchers in Germany have identified a SOX9-responsive regulatory route to IRF6, a major orofacial-cleft risk gene, in ...
Sofi-cel is an allogeneic, off-the-shelf, CD7-targeted CAR-T cell therapy being developed for T-cell cancers. Allotera uses ...
A portable CRISPR-Cas13 assay detected mpox virus with 100% sensitivity and specificity in clinical samples from Sierra Leone. The SHINE test identified as few as 10 copies/µL and delivered results in ...
Base editing offers far more versatility than its name implies. The technique was initially developed to correct ...
A mutation-agnostic CRISPR-Cas strategy that inserts a functional rhodopsin coding sequence into the human RHO locus has ...
CMN founder Jens-Ole Bock will moderate a panel discussion at CSGCT 2026 in Beijing on 20 September on the question that now defines the field: with the first in vivo CRISPR therapy under FDA priority ...
The company’s lead candidate, AXL-003, is advancing towards preclinical evaluation in an autoimmune indication with liver-targeted delivery. This programme builds directly on results showing that AXL ...
On 28 January 2026, nChroma Bio dosed the first participant in its Phase 1/2 clinical trial of CRMA-1001, an investigational CRISPR-Cas-based epigenetic silencer targeting chronic hepatitis B virus ...
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