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

Crispr Therapeutics AG has revenue declines but holds strong financials. Promising Sickle Cell therapy & 2025 data readouts ...

The BEAM-101 edit is designed to inhibit the transcriptional repressor BCL11A from binding to the promoter without disrupting BCL11A expression, leading to increased production of non-sickling and ...

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 ...

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 ...

Ongoing launch of CASGEVY® continues to gain momentum; new cell patient collection initiations expected to significantly ...

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 ...