The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

It uses CRISPR/Cas9 to make a patient’s haematopoietic stem cells produce high levels of foetal haemoglobin (HbF) in red blood cells, by introducing a gene known as BCL11A which down-regulates ...

Ongoing launch of CASGEVY ® continues to gain momentum; new cell patient collection initiations expected to significantly grow in 2025- -More than 50 authorized treatment centers (ATCs) activated ...

Casgevy enables CRISPR/Cas-9 to edit the BCL11A gene in stem cells to produce fetal hemoglobin. The edited genes are infused into a patient's bone marrow to start producing red blood cells with high ...

CRISPR Therapeutics is developing CTX112 for ... cells are edited at the erythroid specific enhancer region of the BCL11A gene. This edit results in the production of high levels of fetal ...

To remedy sickle cells, CRISPR-Cas9 is used to edit genes that inhibit the production of BCL11A, which reactivates rich fetal hemoglobin production to reduce the sickling of blood cells.

CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited ...