iduronicrin genleukocel-T (ISP-001) / Immusoft - LARVOL DELTA

Autologous human peripheral blood B cells genetically engineered to express human iduronidase: Results from a first-in-human clinical trial in subjects with mucopolysaccharidosis type I (MPS I) (ASGCT 2025) - "The first subject is a 25-year-old male who has been on enzyme replacement therapy (ERT; laronidase) for 13 years. These early results support both the dose escalation of ISP-001 to treat MPS I and the expansion of this new engineered B cell platform to treat other human diseases. Disease Focus of Abstract:Storage/Lysosomal Disorders"

Clinical • P1 data • Bone Marrow Transplantation • Hurler Syndrome • Orthopedics • Rare Diseases • IDUA

ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I (clinicaltrials.gov) - P1 | N=2 | Recruiting | Sponsor: Immusoft of CA, Inc. | Trial completion date: Oct 2024 ➔ Jun 2039 | Trial primary completion date: Apr 2024 ➔ Jun 2025

Trial completion date • Trial primary completion date • Hurler Syndrome

ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I (clinicaltrials.gov) - P1 | N=2 | Recruiting | Sponsor: Immusoft of CA, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Hurler Syndrome

ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I (clinicaltrials.gov) - P1 | N=2 | Not yet recruiting | Sponsor: Immusoft Corporation

New P1 trial • Hurler Syndrome