Clinical Trial Update: Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II [Phase 1, Phase 2]

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Summary

An ascending-dose study of the ZFN genome-editing therapy SB-913 in MPS II (Mucopolysaccharidosis II) was terminated; results have been posted.

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Diseases / indications

• Mps Ii
• Mucopolysaccharidosis Ii

Drugs mentioned

• SB-913

Snippet

Sponsor: Sangamo Therapeutics | Conditions: Mucopolysaccharidosis II, MPS II | Phase 1, Phase 2 | Status: TERMINATED | Results posted: 2022-09-27