Mucopolysaccharidosis Type I clinical trials at UCSF

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

  Sorry, in progress, not accepting new patients

  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.

  Oakland, California

• In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases

  Sorry, not yet accepting patients

  The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.

  San Francisco, California

Last updated: January 11, 2021