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 ...

Scribe Therapeutics is preparing to take its first cardiometabolic programme into the clinic in mid-2026, positioning STX-1150 as a potentially durable alternative to today’s chronic lipid-lowering ...

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 ...

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 ...

A hybrid CRISPR design pipeline improves the predictability of genome editing outcomes in mouse embryos. By favouring microhomology-mediated end joining (MMEJ) and validating guides in stem cells, the ...

A genome-wide CRISPR-Cas9 screen to identify transcription factor SOX2 as a critical regulator of PROM1, the gene encoding CD133, in glioblastoma stem cells. Knocking out SOX2 reduced CD133 expression ...

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 ...

Azer-cel (azercabtagene zapreleucel) is an investigational, allogeneic, off-the-shelf CAR T-cell therapy designed to target CD19-expressing B-cell malignancies. It is manufactured from healthy donor T ...

PSMA-UCAR T (BRL-302) is an investigational allogeneic (“universal”) CAR-T cell therapy being evaluated for the treatment of refractory metastatic castration-resistant prostate cancer (CRPC). The ...

Wave Life Sciences has reached alignment with the US Food and Drug Administration on a registrational pathway for WVE-006, a GalNAc-conjugated RNA... Rival Editors Converge on AATD as Data Mount Alpha ...