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