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 ...

Screening ionisable lipids with a gene-editor-sized RNA identified LC-1, an LNP component that retained potency as cargo length increased. In mice, route-specific LC-1 formulations delivered ...

Researchers built a three-layer human blood-vessel model that shows how fibroblasts amplify the vascular pathology of Hutchinson-Gilford progeria... Clinical: FDA Backs Single-Trial Pathway for ...

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 ...

Merck & Co. will present new single-cell functional genomics data generated using purpose-built assays developed with Mission Bio’s Assay Services team. Incyte will share updated clinical single-cell ...

EGEN-5784 is an investigational genetically engineered porcine liver being developed as an extracorporeal support therapy for patients with acute-on-chronic liver failure (ACLF). The donor pig genome ...

PM647 is an investigational, one-time in vivo prime-editing therapeutic candidate being developed for alpha-1 antitrypsin deficiency (AATD). The therapy is designed to correct the disease-causing ...

BEAM-103 is an investigational anti-CD117 monoclonal antibody being developed as a non-genotoxic conditioning agent for use with Beam Therapeutics’ ESCAPE stem-cell therapy platform in sickle cell ...