Product
OTL-200
Aliases
Previously GSK2696274
3 clinical trials
3 indications
Indication
Lysosomal Storage DiseasesIndication
Metachromatic LeukodystrophyIndication
Lysosomal Storage DiseaseClinical 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 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