Signal
7 articles covering "gene-editing"
The RUBY trial of renizgamglogene autogedtemcel (reni-cel), a one-time CRISPR-Cas12a gene-editing therapy, showed that 27 of 28 patients with severe sickle cell disease had no painful crises after treatment, with hemoglobin levels normalizing to 13.8 g/dL by six months.
Researchers at the University of California San Diego have developed pPro-MobV, a second-generation CRISPR-based gene drive that spreads through bacterial populations via conjugal transfer to disable antibiotic resistance genes, demonstrating effectiveness even within biofilms.
Precision BioSciences receives FDA Fast Track for PBGENE-DMD, a gene-editing therapy that excises faulty exons to restore near full-length dystrophin in up to 60 percent of Duchenne patients.
A Nature paper describes INSTALL, a gene editing platform using circular single-stranded DNA to insert therapeutic genes without triggering fatal immune responses, keeping mice healthy at doses where conventional DNA proved lethal.
Gene-edited pancreatic islet cells transplanted into a patient with type 1 diabetes continue producing insulin 14 months later with no immunosuppressive drugs, advancing the case for a functional cure.
Beam Therapeutics' BEAM-302 has achieved the first-ever in-vivo base editing correction of a disease-causing mutation in human patients, reaching therapeutic AAT levels in a Phase 1/2 trial for alpha-1 antitrypsin deficiency. A pivotal Q1 2026 data readout is now imminent.
The FDA's new Plausible Mechanism Pathway enables approval of one-patient gene editing therapies without traditional clinical trials, inspired by the first personalized CRISPR cure for an infant with a fatal metabolic disorder — but ethicists warn of a regulatory Pandora's box.