Back To: Home

CLICK HERE FOR WHAT'S NEW IN:
 




 

ABO-102 shows initial safety, efficacy
September 2019
SHARING OPTIONS:

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.

Back



PAGE UTILITIES


CONTACT US
DDNEWS
Published by Old River Publications LLC
19035 Old Detroit Road
Rocky River, OH USA 44116
Ph: 440-331-6600  |  Fax: 440-331-7563
 
© Copyright 2019 Old River Publications LLC. All righs reserved.  |  Web site managed and designed by OffWhite.