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Sleeping Beauty engineered plasmablasts for Hurler syndrome
NCT04284254Phase 2Withdrawn
- Sponsor
- Masonic Cancer Center
- Start
- Dec 2022
- Primary completion
- Jun 2023
- Modality
- Gene Editing, Gene Silencing, Cell Therapies
01
Overview
Phase 2, open-label trial in mucopolysaccharidosis type I, sponsored by Masonic Cancer Center. Primary endpoint: Maximum Tolerated Dose at 1 year. Planned start Dec 2022; status: withdrawn.
02
Conditions
mucopolysaccharidosis type I
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