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Flanigan, Kevin M.; Vetter, Tatyana A.; Simmons, Tabatha R.; Iammarino, Megan; Frair, Emma C.; Rinaldi, Federica; Chicoine, Louis G.; Harris, Johan; Cheatham, John P.; Cheatham, Sharon L.; Boe, Brian; Waldrop, Megan A.; Zygmunt, Deborah A.; Packer, Davin; Martin, Paul T.
Molecular therapy. Methods & clinical development, 12/2022, Volume: 27Journal Article
In a phase 1/2, open-label dose escalation trial, we delivered rAAVrh74.MCK.GALGT2 (also B4GALNT2) bilaterally to the legs of two boys with Duchenne muscular dystrophy using intravascular limb infusion. Subject 1 (age 8.9 years at dosing) received 2.5 × 1013 vector genome (vg)/kg per leg (5 × 1013 vg/kg total) and subject 2 (age 6.9 years at dosing) received 5 × 1013 vg/kg per leg (1 × 1014 vg/kg total). No serious adverse events were observed. Muscle biopsy evaluated 3 or 4 months post treatment versus baseline showed evidence of GALGT2 gene expression and GALGT2-induced muscle cell glycosylation. Functionally, subject 1 showed a decline in 6-min walk test (6MWT) distance; an increase in time to run 100 m, and a decline in North Star Ambulatory Assessment (NSAA) score until ambulation was lost at 24 months. Subject 2, treated at a younger age and at a higher dose, demonstrated an improvement over 24 months in NSAA score (from 20 to 23 points), an increase in 6MWT distance (from 405 to 478 m), and only a minimal increase in 100 m time (45.6–48.4 s). These data suggest preliminary safety at a dose of 1 × 1014 vg/kg and functional stabilization in one patient. Display omitted Results of a first-in-human phase 1/2a clinical trial are reported wherein two patients with Duchenne muscular dystrophy were given GALGT2 gene therapy. The study shows possible stabilization of muscle function in one of two subjects, who was treated with a higher dose and at a younger age.
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