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Bluebird bio gene therapy shows sustained benefit in rare metabolic disorder

August 31, 2020

Bluebird bio (BLUE +2.0%) announces long-term results from a Phase 2/3 clinical trial, STARBEAM, evaluating gene therapy elivaldogene autotemcel (eli-cel, Lenti-D) in patients with cerebral adrenoleukodystrophy (CALD), a rare inherited metabolic disorder in which very long chain fatty acids build up in the brain damaging nerve cells.

Median follow-up was 30.0 months (range: 9.1 – 70.7 months) in 32 patients. 20 of the 32 have enrolled in a long-term follow-up study Nine others will continue to be followed in STARBEAM (not yet reached 24 months post-treatment) and two withdrew.

87% (n=20/23) of treated patients who have or would have reached month 24 of follow-up achieved the primary endpoint of being alive and free of six major functional disabilities (MFDs) at month 24. 14 subjects have at least four years’ follow-up while 10 have reached five years.

97% (n=31/32) experienced stable neurologic function score (NFS) after treatment with eli-cel, including 24 with NFS of 0 (no concerns with neurologic functions).

A 35-subject Phase 3 trial, ALD-104, is underway with an estimated completion date in February 2024.

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