Organization
Orchard Therapeutics
4 clinical trials
Clinical trial
An Open Label, Non-randomized Trial to Evaluate the Safety and Efficacy of a Single Infusion of OTL-200 in Patients With Late Juvenile (LJ) Metachromatic Leukodystrophy (MLD).Status: Active (not recruiting), Estimated PCD: 2025-03-31
Clinical trial
A Multi-center, Randomized, Active Controlled Clinical Trial to Evaluate the Efficacy and Safety of OTL-203 in Subjects With Mucopolysaccharidosis Type I, Hurler Syndrome (MPS-IH) Compared to Standard of Care With Allogeneic Hematopoietic Stem Cell Transplantation (Allo-HSCT)Status: Recruiting, Estimated PCD: 2028-03-01
Clinical trial
A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic LeukodystrophyStatus: Active (not recruiting), Estimated PCD: 2018-04-09
Clinical trial
A Single Arm, Open Label, Clinical Study of Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Containing Human ARSA cDNA (OTL-200), for the Treatment of Early Onset Metachromatic Leukodystrophy (MLD)Status: Active (not recruiting), Estimated PCD: 2022-04-06