Clinical Trial: ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
Study Status: Completed
Recruit Status: RECRUITING
Condition: Mucopolysaccharidosis IH/S
Study Type: INTERVENTIONAL
Official Title: A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Adult Patients With Mucopolysaccharidosis Type I Hurler-Scheie and Scheie
Brief Summary:
A first-in-human study using ISP-001 in adult patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
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