bluebird bio announces further advances in gene therapy for patients with ALD

ALD-104 is now open for enrolment! More information on this clinical study can be found at
ALD-104 is an open-label, single-arm, multi-centre study aimed at evaluating safety and efficacy of bluebird bio’s investigational gene therapy after myeloablative conditioning using busulfan and fludarabine in paediatric patients with cerebral adrenoleukodystrophy (CALD).