Mucopolysaccharidosis I

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I

A Phase I, Multicenter, Open-label, Single-dose, Dose-ranging Study to Assess the Safety and Tolerability of SB-318, a rAAV2/6-based Gene Transfer in Subjects With Mucopolysaccharidosis I (MPS I)

About: The purpose of the study is to evaluate the safety, tolerability of ascending doses of SB-318. SB-318 is an intravenously delivered Zinc Finger Nuclease (ZFN) Therapeutic for genome editing. It inserts a correct copy of the α-L-iduronidase (IDUA) gene into the Albumin locus in hepatocytes with the goal of lifelong therapeutic production of the IDUA enzyme.

Ages Eligible for Study: 18 Years and older (Adult, Senior)

Sexes Eligible for Study: All

Status:  This study is ongoing, but not actively recruiting and enrolling participants. Recruitment and enrollment may resume in the future.

For more information about this study, including eligibility criteria, please go to the studies page. If you are interested in participating in this study or have questions about research, please contact Gee Kim, Clinical Research Coordinator.