U.S., July 2 -- ClinicalTrials.gov registry received information related to the study (NCT07046338) titled 'Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD' on June 23.
Brief Summary: This is a Phase I/II clinical trial of gene therapy for treating Metachromatic leukodystrophy (MLD) using a safety and efficacy improved self-inactivating lentiviral vector TYF-ARSA to transduce patient-derived hematopoietic stem cells (HSCs), with the goal of achieving therapeutic gene correction through transplantation of genetically modified HSCs. The primary objectives are to evaluate the safety and efficacy of the gene therapy clinical protocol.
Study Start Date: June 01
Study Type: INTERVENTIONAL
Condition:
Metachromatic Leukodystrophy (MLD)...