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Abeona Therapeutics Announces Positive Interim Data from MPS III Gene Therapy Programs Presented at WORLDSymposium™
Neurocognitive development of young MPS IIIA patients preserved up to two years post ABO-102 treatment Dose-dependent and sustained reductions in
About this update from Abeona Therapeutics Inc.
[{"type":"text","content":"Neurocognitive development of young MPS IIIA patients preserved up to two years post ABO-102 treatment\n Dose-dependent and sustained reductions in disease-specific biomarkers denotes clear biologic effects of ABO-102 and ABO-101 First patient treated in cohort 3 of ABO-101 MPS IIIB trial; total enrollment eight patients Favorable safety profile observed in both studies NEW YORK and CLEVELAND, Feb. 12, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that researchers from the Abigail Wexner Research Institute (AWRI) at Nationwide Children’s Hospital presented positive interim data from two ongoing Phase I/II clinical trials evaluating ABO-102 and ABO-101, the Company’s investigational gene therapies for MPS IIIA and MPS IIIB, respectively, at WORLDSymposium™. Results from the Transpher A study demonstrated that MPS IIIA patients younger than 30 months treated with ABO-102 in dose cohort 3 continue to show neurocognitive development 18 months to two years after treatment. Reductions in cerebrospinal fluid (CSF) heparan sulfate (HS), denoting enzyme activity in the central nervous system, and liver volume reductions remain stable two years after treatment. Results from the Transpher B study showed that ABO-101 also improved multiple disease biomarkers providing clear evidence of a biologic effect in patients with MPS IIIB. Dosing in cohort 2 is complete and the first patient in cohort 3 was treated in late January, with a total of 8 patients treated to date. Both therapies have been well-tolerated to date. Abeona licensed the AAV9-based gene therapy technology underpinning ABO-102 and ABO-101 from AWRI at Nationwide Children’s where it was developed. Today’s presentations are available on abeonatherapeutics.com by following this link: https://investors.abeonatherapeutics.com/news-events “In total, the new results continue to show that early treatment with ABO-102 can help preserve neurodevelopment in children with MPS IIIA. These data will inform our ongoing discussions with the FDA and EMA, as we work towards providing a regulatory update in the second quarter,” said João Siffert, M.D., Chief Executive Officer. “For ABO-101, the reductions in disease-specific biomarkers are encouraging and demonstrate a clear biologic effect, which parallels that...