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ABO-102 shows initial safety, efficacy
September 2019
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NEW YORK & CLEVELAND—Late July saw Abeona Therapeutics Inc. share positive data from its Phase 1/2 clinical trial of ABO-102, a one-time, adeno-associated virus 9 gene therapy for Sanfilippo syndrome type A. The data show that the youngest patients in the study, who were enrolled in the high-dose cohort 3, all saw preservation of neurocognitive development 12 to 18 months after treatment with ABO-102. There was noticeable, sustained improvement in biomarkers as well, and longer-term safety was favorable eight months to two years after treatment. Dose-dependent reductions in CSF heparan sulfate were seen in all three cohorts, and no product-related serious adverse events have been reported to date. ABO-102 is a novel gene therapy delivered via one-time IV infusion, and delivers a functional copy of the SGSH gene to cells in the central nervous system and peripheral organs.

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