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 ...

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 cranial neural crest cells rather than only in oral epithelium. CRISPR-Cas9 ...

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 ...

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 ...

CG101 is an investigational off-the-shelf allogeneic iPSC-derived NK-cell therapy for acute myeloid leukaemia (AML). The cells are generated from induced pluripotent stem cells and engineered using ...

This is a Long-term Follow-up (LTFU) study in patients who received BEAM-101 in the parent study (Study BTX-AUT-001). Eligible patients who received BEAM-101 will be asked to participate in this LTFU ...

FT839 is an investigational, off-the-shelf iPSC-derived dual-CAR T-cell therapy designed to target both CD19 and CD38 for the treatment of complex autoimmune diseases and potentially haematological ...