Shots:

• The BLA is based on the P-II/III (Starbeam/ALD-102) study to evaluate Eli-cel in 32 patients aged ≤18yrs. with CALD without a matched sibling donor. Additionally, BLA contains data from 23 patients dosed in the P-III (ALD-104) study
• The trial met its 1EPs i.e., patients achieved MFD-free survival (90.6%) @24mos., 2 patients withdrew from the study & 1 additional patients experienced RDP resulted in MFDs & death while the median duration of follow-up is 3.5yrs. (42.3mos.). Upon completion of an (ALD-102) study, patients will enroll in follow-up study LTF-304
• If approved, Eli-cel will be 1st approved gene therapy & is designed to treat the irreversible ND & stabilize neurologic function

Ref: Bluebird Bio | Image: Bluebird bio