Thousands of Californians live with sickle cell disease, a painful blood disorder. But new treatments, including a promising form of gene therapy, are offering hope to ...

A one-time gene-editing therapy, exagamglogene autotemcel (exa-cel), has demonstrated sustained and clinically meaningful ...

Gene therapy for sickle cell disease may help improve a major contributing factor to stroke risk in patients, reports a new study from St. Jude Children's Research Hospital. Many people with sickle ...

Fifteen-year-old Jayden Wilsey of Falmouth is the first person at Boston Children's Hospital to receive human gene therapy for sickle cell disease, following approval of the therapy in 2024 by the U.S ...

Exagamglogene autotemcel (exa-cel) significantly improved HRQOL in severe SCD patients, eliminating severe VOCs and hospitalizations in most cases. The CLIMB SCD-121 trial showed sustained ...

CHICAGO (WLS) -- Sickle cell anemia disproportionately affects Black people, causing severe pain, potential organ damage and in some cases, death. Many times, only blood transfusions can provide ...

MOBILE, Ala. (WALA) - Gene therapy for sickle cell disease (SCD) is a cutting-edge treatment that targets the root cause of the disorder—a genetic mutation in the HBB gene, which leads to the ...

An increasing demand for gene therapies is focusing the industry on enhancing production capabilities, transitioning from ...

Life expectancy has soared and interest in the disease is growing, but breakthrough gene therapies are extraordinarily ...

Treatment with exagamglogene autotemcel (exa-cel) led to robust and sustained improvements in quality of life for patients with severe sickle cell disease (SCD) or transfusion-dependent beta ...

Exagamglogene autotemcel (exa-cel), a CRISPR/Cas9 gene-edited cell therapy, eliminated vaso-occlusive crises in 97% of participants with severe sickle cell disease. The therapy led to substantial ...

Public health experts have emphasised the urgent need for Nigeria to adopt life-changing gene therapy for the treatment of sickle cell disease. Official data shows that approximately 4 million ...