OTL-200

GENETIC2 trials

Sponsors

Orchard Therapeutics

Conditions

Lysosomal Storage DiseaseLysosomal Storage DiseasesMetachromatic Leukodystrophy

Phase 2

A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD)

CompletedNCT03392987

Orchard TherapeuticsLysosomal Storage Disease, Metachromatic Leukodystrophy

Start: 2018-01-25End: 2026-03-10Updated: 2026-03-16

Phase 3

OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)

Active, not recruitingNCT04283227

Orchard TherapeuticsLysosomal Storage Diseases, Metachromatic Leukodystrophy

Start: 2022-01-17End: 2031-03-31Updated: 2025-09-05

Related Papers

Long-Term Effects of Atidarsagene Autotemcel for Metachromatic Leukodystrophy

New England Journal of Medicine2025-04-2312 citations

Monitoramento do Horizonte Tecnológico como ferramenta de sustentabilidade: atidarsagene autotemcel

Jornal de Assistência Farmacêutica e Farmacoeconomia2024-11-30

Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access.

2022-01-01191 citations