why choose us

First received: December 12, 2022

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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