Despite progress in healthcare services for individuals living with sickle cell disease (SCD) in Africa, substantial gaps remain in advanced treatments for SCD. To help address this burden, Tanzania ...

Children's Hospital Los Angeles added a 10th cell and gene therapy treatment for children, adolescents and young adults with serious medical conditions. The newest gene therapy treatment, Casgevy ...

The company, which secured $200 million from investors in a private funding deal last week, learned Tuesday of a serious side effect experienced by one participant on a high dose of its Rett therapy.

1 Department of Pediatrics, West China Second University Hospital, Sichuan University, Chengdu, China 2 Key Laboratory of Birth Defects and Related Diseases of Women and Children, Ministry of ...

That is, until last December, when the FDA approved “two milestone treatments,” Casgevy and Lyfgenia, the first gene therapies for the treatment of SCD in patients 12 years and older. Within months, ...

Bluebird Bio’s Lyfgenia is listed at $3.1 million. Neither includes the cost of care to stay in the hospital or for chemotherapy. To manage the hefty price tag, insurers have implemented ...

The grant from the U.S. Department of Defense was given to the study’s lead researcher Ankit A. A month after its U.S. approval, Lyfgenia (lovotibeglogene autotemcel), Bluebird Bio’s gene therapy for ...

This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. As 2025 begins, emerging biotechnology firms can ...

Bluebird bio is a case study. The company has three approved gene therapies: Zynteglo for beta thalassemia, Skysona for cerebral adrenoleukodystrophy and Lyfgenia for sickle cell. Yet the firm has ...