Abeona Therapeutics (NASDAQ:ABEO) is up 11% premarket on light volume on the heels of positive data from its Phase 1/2 clinical trial evaluating gene therapy ABO-102 in patients with Sanfilippo syndrome type A (MPS IIIA), a rare inherited lysosomal storage disorder in which sugar molecules build up in cells leading to development disabilities.
The youngest patients enrolled in cohort 3 showed preservation of neurocognitive development 12-18 months after treatment. There was also “robust and sustained” improvement in certain biomarkers of biological effect.
No new safety signals have been reported.
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