NCT03041324 - Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

May 11, 2017

Primary Completion Date

May 7, 2021

Study Completion Date

May 7, 2021

Conditions

Mucopolysaccharidosis IIMPS II

Interventions

BIOLOGICAL

SB-913

Single dose of each of the 3 components of SB-913: ZFN1, ZFN2 and hIDS Donor

Trial Locations (5)

NYU School of Medicine, Neurogenetics Division, New York

University of North Carolina, Chapel Hill

Cincinnati Children's Hospital Medical Center, Cincinnati

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago

UCSF Benioff Children's Hospital Oakland, Oakland