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

First-in-human clinical trial of autologous human B cells engineered to express human iduronidase in subjects with MPS I: Support for pediatric studies (ASGCT 2026) - "These results support the further development of ISP-001 in adult attenuated MPS I patients, with approval to expand the trial to include pediatric attenuated patients to address both dysostosis multiplex and neurological disease. This work was funded in part by the California Institute for Regenerative Medicine."

Clinical • First-in-human • P1 data • Bone Marrow Transplantation • CNS Disorders • Hurler Syndrome • Orthopedics • Pediatrics • Rare Diseases • IDUA

ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I (clinicaltrials.gov) - P1 | N=11 | Recruiting | Sponsor: Immusoft of CA, Inc. | Trial completion date: Jun 2039 ➔ Jun 2044 | Trial primary completion date: Jun 2025 ➔ Jun 2029

First-in-human • Trial completion date • Trial primary completion date • Hurler Syndrome

Immusoft Receives FDA Fast Track Designation for ISP-001, a First-in-Class Engineered B Cell Therapy for MPS I (PRNewswire)

Fast track • Lysosomal Storage Diseases

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