The US FDA has accepted Intellia Therapeutics’ application for lonvoguran ziclumeran (lonvo-z, formerly NTLA-2002) and ...

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 ...

Delivering CRISPR-Cas9 as mRNA in lipid nanoparticles preserved the fitness and erythroid potential of edited human ...

Global adoption growing with more than 400 institutions across 33 countries ordering DNA constructs up to 49.1 kb, with ...

(Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta CAR T cell and in ...

–ARIA investment advances QBI's landmark Science study, accelerating the path from molecular discovery to new precision ...

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 ...

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... Redesigned Landing Sites Lift Bxb1 Integration ...

Three boys with Duchenne muscular dystrophy retained broadly stable motor function one year after receiving GEN6050X, an experimental base-editing and gene-addition therapy. Cardiac and pulmonary ...

A new artificial intelligence–guided strategy can design highly divergent yet functional variants of TnpB, a compact RNA-guided nuclease that is evolutionarily related to CRISPR-Cas12 enzymes. By ...

A search for new CRISPR enzymes to improve crop breeding has unexpectedly led to a new strategy for selectively eliminating cancer cells. The story highlights how discoveries originating in ...

EPI-321 is designed to treat the underlying cause of FSHD by durably repressing expression of the DUX4 gene, whose inappropriate activation drives muscle degeneration. Rather than editing DNA, the ...