Major is one of the first pediatric patients in Florida to receive gene therapy for sickle cell disease since the FDA ...

Caden Major was born with sickle cell disease – an inherited blood disorder affecting red blood cells. Now at 19 years old, ...

In the big picture, matched sibling donor transplants remain the standard of care, Boelens said. But gene therapy is ...

Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an ...

Key Highlights Motixafortide effectively mobilized sufficient hematopoietic stem cells (HSCs) in patients with sickle cell disease enabling accelerated access to gene therapies 90% (9 of 10) of ...

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 ...

About 100,000 people in the U.S. have sickle cell disease, a condition that primarily affects people of color, the CDC says.

The National Institute for Health and Care Excellence (NICE) has issued final draft guidance recommending a CRISPR gene editing-based treatment for severe sickle cell disease (SCD). The regulator said ...

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 ...

A study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers ...

LOS ANGELES (KABC) -- It's been more than a month since the FDA approved two milestone gene-editing treatments for sickle cell disease, but lining up patients for these therapies will be a challenge ...

The chief medical officer of Genetix Biotherapeutics, formerly known as Bluebird bio, discusses timelines and operational ...