COMPANY

Orchard Therapeutics

HQ: London, United Kingdom source · orchard-tx.com ↗

FDA-approved therapeutic company Clinical Phase 3

Keywords: gene therapy Metachromatic Leukodystrophy Lysosomal Storage Disease Mucopolysaccharidosis IH MPS-IH (Hurler Syndrome) Lysosomal Storage Diseases OTL-200 OTL-200 Gene Therapy

Location: London, United Kingdom · source · Oct 13, 2026

Profile checked Oct 10, 2026

5Trials (company is sponsor)

Timeline

Dec 11, 2023 Trial start Phase 3: A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT source ↗
Jan 17, 2022 Trial start Phase 3: OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD) source ↗
May 11, 2018 Trial start Phase 1/Phase 2: Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant source ↗
Jan 25, 2018 Trial start Phase 2: A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD) source ↗
Apr 9, 2010 Trial start Phase 1/Phase 2: Gene Therapy for Metachromatic Leukodystrophy (MLD) source ↗

Upcoming milestones

2028-03 (ESTIMATED) · Phase 3 · A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT · NCT06149403
2035-01 (ESTIMATED) · Phase 1/Phase 2 · Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant · NCT03488394

Estimated primary completion per ClinicalTrials.gov. Dates move.

Clinical trials (lead sponsor) 5

Record	Title	Phase	Status	Start	Primary completion
NCT03488394	Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant	Phase 1/Phase 2	Active, not recruiting	May 11, 2018	2035-01 (estimated)
NCT03392987	A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD)	Phase 2	Completed	Jan 25, 2018	Apr 6, 2022 (actual)
NCT06149403	A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT	Phase 3	Active, not recruiting	Dec 11, 2023	2028-03 (estimated)
NCT01560182	Gene Therapy for Metachromatic Leukodystrophy (MLD)	Phase 1/Phase 2	Completed	Apr 9, 2010	Apr 9, 2018 (actual)
NCT04283227	OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)	Phase 3	Active, not recruiting	Jan 17, 2022	Jan 31, 2026 (estimated)