BLUE - Bluebird bio gains 4% on promising eli-cel data in rare metabolic brain disorder

Bluebird bio (BLUE) announces new data from the clinical development program for its its elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy in patients with cerebral adrenoleukodystrophy ((CALD)), a rare, X-linked metabolic disorder. Starbeam Study (ALD-102)/Long-Term Follow-Up Study (LTF-304)The results show that at 24 months of follow-up, 90% of patients (27/30) in the study of eli-cel (ALD-102) met the primary endpoint and were alive and free of major functional disabilities (MFDs) at two years of follow-up.Patients in long-term follow-up study (LTF-304) continue to remain alive and MFD-free through up to nearly seven years (up to 82.7 months) of follow-up, suggesting eli-cel stabilizes the progression of disease. Of the 32 patients treated, 31 had stable neurologic function score ((NFS)) at last available visit following treatment with eli-cel, and 23 maintained an NFS of 0 which indicates that there is no observed impairment in the neurologic functions.Phase 3 ALD-104 Study17 of 19 evaluable patients achieved neutrophil engraftment and

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Bluebird bio gains 4% on promising eli-cel data in rare metabolic brain disorder