FDA committee unanimously endorses gene therapy for cerebral adrenoleukodystrophy

Biotechnology company bluebird bio announced that an FDA advisory committee has unanimously endorsed its gene therapy treatment for early active cerebral adrenoleukodystrophy.
According to a company press release, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee voted 15 to 0 that the benefits of bluebird bio’s elivaldogene autotemcel (eli-cel) therapy outweighed the risks for patients younger than 18 years without an available and willing human leukocyte antigen-matched sibling hematopoietic stem cell donor.
Early active cerebral adrenoleukodystrophy (CALD) is a

Biotechnology company bluebird bio announced that an FDA advisory committee has unanimously endorsed its gene therapy treatment for early active cerebral adrenoleukodystrophy.
According to a company press release, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee voted 15 to 0 that the benefits of bluebird bio’s elivaldogene autotemcel (eli-cel) therapy outweighed the risks for patients younger than 18 years without an available and willing human leukocyte antigen-matched sibling hematopoietic stem cell donor.
Early active cerebral adrenoleukodystrophy (CALD) is a