ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I

A first-in-human study using ISP-001 in adult patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.

Inclusion Criteria:Diagnosis of Mucopolysaccharidosis type I Hurler-Scheie or Scheie syndrome.Age ≥ 18 years at time of study registration.Creatinine clearance, calculated or measured directly, that is >60ml/min/1.73m2.Ejection fraction ≥ 40% by echocardiogram.Must commit to traveling to the study site for the necessary follow-up evaluations.Must agree to stay <45-minute drive from the study site for a minimum of 5 days after cell infusion.Exclusion Criteria:Known familial inherited cancer syndrome. Suspected cases will be investigated, per the physicians discretion, using relevant genetic tests to determine presence of germline mutations.History of B cell related cancer, EBV lymphoproliferative disease or autoimmune disorders.Evidence of active graft-vs-host disease.Underwent a previous hematopoietic stem cell transplant (HSCT).Requirement for systemic immune suppression.Requirement for continuous supplemental oxygen.Any medical condition likely to interfere with assessment of safety or efficacy of the study treatment.In the investigator's judgement, the subject is unlikely to complete all protocol-required study visits or procedures, including follow up visits, or comply with the study requirements for participation.Other protocol defined inclusion/exclusion criteria may apply.