This site is intended for healthcare professionals
News

FDA Advisory Committee unanimously recommends eli-cel, an investigational LVV gene therapy for the treatment of cerebral adrenoleukodystrophy.- bluebird bio

Read time: 1 mins
Published:11th Jun 2022

On June 9, the FDA's Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) unanimously endorsed eli-cel, an investigational LVV gene therapy for the treatment of cerebral adrenoleukodystrophy, in a 15 to 0 vote.

 

The childhood form of cerebral adrenoleukodystrophy (Lorenzo's Oil disease) is an appallingly devastating neurodegenerative genetic disease that affects boys and is generally fatal if left untreated. Allogeneic hematopoietic stem cell transplant (allo-HSCT) is currently the only available effective therapy, but is potentially associated with serious safety risks, including graft rejection, graft-versus-host disease and transplant-related mortality. The Lenti-D gene therapy product candidate consists of a patient’s own modified hematopoietic stem cells and should avoid the problems of immune-incompatibility that can complicate allo-HSCT.

Condition: Cerebral Adrenoleukodystrophy
Type: drug

Learning Zones

The Learning Zones are an educational resource for healthcare professionals that provide medical information on the epidemiology, pathophysiology and burden of disease, as well as diagnostic techniques and treatment regimens.