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Abeona Therapeutics Says Sanfilippo Syndrome Type A Trial Data Shows ‘Robust, Durable Clinical Effects’


Abeona Therapeutics (ABEO) gained almost 3% in pre-market trade Friday after the company said its trial of ABO-102 in Sanfilippo syndrome Type A demonstrated a “durable and significant reduction” of underlying disease pathology across multiple clinical measures.

Data from cohort one, which the company released Friday, showed robust and durable clinical effects achieved one year post-administration, with significant reductions in biopotency and biophysical measures, preservation of deep brain architecture, and stabilization across multiple neurocognitive assessments reported in comparison to untreated control subjects, the company said.

“We remain encouraged by signs of tolerability and biological effects that we have observed in Cohort 1 one year post-injection,” stated Juan Ruiz, chief medical officer. “Importantly, the stabilization of deep brain architecture at one year highlights the potential of intravenous route of delivery in accessing the basal ganglia to promote neural stabilization. We are pleased to see continued decreases in CSF HS in the Cohort one year post-administration, along with positive signs of neurocognitive and physical benefits at the low dose.”