Orchard Therapeutics has announced initial results from its gene therapy in development for the treatment of metachromatic leukodystrophy (MLD).
The therapy, a cryopreserved formulation of OTL-200, demonstrated that cellular engraftment using a cryopreserved formulation is similar to that observed using a fresh formulation, with the longest patient having 12 months of follow-up since treatment.
The company also confirmed that the emerging safety profile of the cryopreserved formulation was consistent with the safety profile observed in the fresh formulation, and that the preliminary evidence also suggests that the first patient treated with the cryopreserved formulation has acquired motor capabilities consistent with the physiologically progressive acquisition of new motor skills observed in healthy children.
“We are pleased that these initial data suggest that using gene-corrected cells that have been cryopreserved has a similar impact on clinical biomarkers for early-onset MLD patients as the OTL-200 fresh formulation,” said Dr. Valeria Calbi, a haematologist at San Raffaele Scientific Institute and SR-Tiget and an investigator of the study.
“The four treated patients showed good levels of engraftment of gene-corrected cells and reconstitution of ARSA activity at multiple time points, as well as encouraging early trends in GMFM scores that we look forward to evaluating with additional follow-up. We believe that these data further support the positive benefit / risk profile of OTL-200 as a therapy with potential lifelong benefit for patients with MLD.”
MLD is a devastating and rapidly progressing disease with no standard treatment options. In its most severe forms of the rare and life-threatening inherited disease, patients will not survive beyond their first decade of life.