SB-318

BIOLOGICAL2 trials

Sponsors

Sangamo Therapeutics

Conditions

Hemophilia BMPS IMucopolysaccharidosis IMucopolysaccharidosis II

Phase 1

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

TerminatedNCT02702115

Sangamo TherapeuticsMPS I

Start: 2017-05-24End: 2021-11-03Updated: 2023-01-26

Unknown Phase

Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX

TerminatedNCT04628871

Sangamo TherapeuticsHemophilia B, Mucopolysaccharidosis I, Mucopolysaccharidosis II

Start: 2020-11-03End: 2026-06-17Updated: 2026-08-13

Related Papers

A Highly Efficacious PS Gene Editing System Corrects Metabolic and Neurological Complications of Mucopolysaccharidosis Type I.

2020-04-0847 citations

CRISPR/Cas9: A Potential Life-Saving Tool. What’s next?

Molecular Therapy — Nucleic Acids2017-12-0111 citations