OTL-201 / Kyowa Kirin - LARVOL DELTA

Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA (clinicaltrials.gov) - P1/2 | N=5 | Active, not recruiting | Sponsor: University of Manchester | Trial completion date: Oct 2024 ➔ Oct 2026

Trial completion date • Gene Therapies • Hunter Syndrome • Lysosomal Storage Diseases

CLINICAL TRIAL UPDATE: EX-VIVO AUTOLOGOUS STEM CELL GENE THERAPY IN MPSIIIA (EBMT 2022) - P1/2 | " These 5 children with MPSIIIA have safely received ex-vivo autologous HSC-GT which was well tolerated, continues to deliver supra-physiological levels of enzyme and reduces substrate accumulation."

Preclinical • Bone Marrow Transplantation • CNS Disorders • Developmental Disorders • Gene Therapies • Hunter Syndrome • Immunology • Inflammation • Lysosomal Storage Diseases • Metabolic Disorders • Pediatrics • Rare Diseases • Transplantation • CD34 • ITGAM

Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type IIIA (clinicaltrials.gov) - P1/2; N=5; Active, not recruiting; Sponsor: University of Manchester; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed • Preclinical • Gene Therapies • Hunter Syndrome • Lysosomal Storage Diseases