OTL-201
/ Kyowa Kirin
- LARVOL DELTA
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March 31, 2025
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
January 30, 2022
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
July 06, 2021
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
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