NHS Scotland has approved a one-time CRISPR gene therapy for severe sickle cell disease, offering durable reduction in vaso-occlusive crises for eligible patients.

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 ...

Under the agreement, participating states will receive “discounts and rebates” from the drugmakers if the treatments don’t ...

A new gene therapy for people who can have a stem cell transplant but do not have a suitable donor has been approved.

We've been hearing a lot of reporting recently about using the latest genetic technology to try and treat sickle cell disease. Scientists are also making progress using older DNA techniques to help ...

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 ...

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 ...

Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment opportunity for sickle cell disease and β-thalassemia. The discovery, ...

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 ...

A free two-day event in Cambridge will unite experts to discuss translational advances in cell and gene therapy, spanning rare diseases, cancer immunotherapy, and genome editing.