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 ...

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 ...

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

This causes BCL11A to not be made which in turn allows gamma globin ... notably sickle cell disease and beta thalassemia. “Importantly it is also likely that making gamma globin is safe – it is our ...

Researchers have been "making great inroads" in precision medicine, and a 15 percent cap on indirect costs could quash the ...

A Thalassemia screening desk has been inaugurated at the NADRA centre in Sukkur on Saturday marking a significant step forwards the fight against the genetic disorder. The desk, established in ...

It means that eligible SCD patients in England now have access to the therapy following the prior agreement for transfusion-dependent beta thalassemia (TDT) patients announced last August.

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® ...

In Guangxi, Hainan, and Fujian Province in southern China, β-thalassemia is a frequent monogenic hereditary disorder that is primarily defined by hemolytic anemia brought on by inefficient ...