Back To: Home : Featured Technology : Gene and Cell Therapies

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
1000 N West Street, Suite 1200,
Wilmington, Delaware, 19801
Ph: 888-781-0328 |  Fax: 705-528-0270
 
© Copyright 2020 Old River Publications LLC. All righs reserved.  |  Web site managed and designed by OffWhite.