Immusoft of CA, Inc.
A first-in-human study using ISP-001 in patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
This is a Phase 1, first-in-human, open-label, single-arm study in which patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie are treated with autologous plasmablasts engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty transposon system (ISP-001). This study will evaluate the safety and tolerability of ISP-001.
Inclusion Criteria: * Diagnosis of Mucopolysaccharidosis type I Hurler-Scheie or Scheie syndrome. * Age ≥ 10 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,…
Autologous plasmablasts (B cells) engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty (SB) transposon system.
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