Vertex Pharmaceuticals (Nasdaq: VRTX) announced today a reimbursement agreement with NHS England for eligible sickle cell disease (SCD) patients to access the CRISPR/Cas9 gene-edited therapy, CASGEVY® ...

Agreement means eligible sickle cell disease (SCD) patients in England now have access to CASGEVY - - Agreement for CASGEVY in transfusion-dependent beta thalassemia (TDT) was previously reached ...

Advances in CRISPR technologies promise to accelerate the burgeoning pipeline of gene-editing therapies and broaden access to these disease-altering therapies.

Detailed price information for Crispr Therapeutics Ag (CRSP-Q) from The Globe and Mail including charting and trades.

CASGEVY is a non-viral ... and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene. This edit results in the production of high levels of fetal hemoglobin ...

January 31, 2025 expert reaction to NICE final draft guidance on exagamglogene autotemcel (exa-cel) for severe sickle cell disease . Scientists comment on final draft guidance fro ...

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 at the erythroid ...

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 ...

To remedy sickle cells, CRISPR-Cas9 is used to edit genes that inhibit the production of BCL11A, which reactivates ... While Vertex Pharmaceuticals took Casgevy to market, CRISPR Therapeutics ...