bluebird bio presents positive results for inherited blood disorder gene therapies

2023-11-06

基因疗法优先审批ASH会议

bluebird bio presents positive results for inherited blood disorder gene therapies

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来源: PMLiVE

bluebird bio has announced updated results from its gene therapy programmes in sickle cell disease (SCD) and beta-thalassaemia (beta-thal).

The results, which will be presented at the American Society of Hematology (ASH) Annual Meeting and Exposition, include long-term follow-up data from the company's lentiviral vector (LVV) gene therapies in SCD patients who have a history of vaso-occlusive events (VOEs) and those with beta-thalassemia who require regular red blood cell transfusions.

Affecting approximately 100,000 people in the US, SCD is a life-long, incurable genetic disease that causes red blood cells to take a distinct crescent shape, which can block blood vessels and affect the way oxygen is carried around the body.

Bluebird’s lovotibeglogene autotemcel (lovo-cel) is an investigational one-time treatment designed to add functional copies of a modified form of the beta-globin gene into a patient’s own haematopoietic stem cells so their red blood cells can produce anti-sickling haemoglobin.

Updated results from 47 lovo-cel-treated patients followed for a median of 35.5 months show that the therapy, which was accepted by the US Food and Drug Administration for priority review earlier this year, was associated with sustained adult haemoglobin production and near-complete resolution of VOEs and severe VOEs, as well as sustained improvements in health-related quality of life.

Beta-thal, another inherited blood disorder, is caused by a gene defect that impairs the ability of red blood cells to produce haemoglobin. Patients with the most severe form of beta-thal develop life-threatening anaemia and have to undergo regular blood transfusions, a lengthy process typically needed every two to five weeks.

Betibeglogene autotemcel (beti-cel), which is already approved in the US under the brand name Zynteglo for adult and paediatric patients with beta-thal who require regular red blood cell transfusions, works in a similar way to lovo-cel to allow patients to make normal-to-near-normal levels of total haemoglobin without regular red blood cell transfusions.

The new results for the therapy set to be presented at ASH demonstrate a sustained transfusion independence after up to nine years of follow-up and improvements in quality of life reported at month 36.

Richard Colvin, bluebird’s chief medical officer, said: “The robustness of this dataset is unparalleled, giving us continued confidence that the transformational impact these therapies may have for patients is sustained over time.

“We especially look forward to the first ever presentation of data from our long-term follow-up study of lovo-cel, the most deeply studied gene therapy in development for SCD.”

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