Sio Gene Therapies Inc (fka SIOX)

[georgejjl]

It appears there are very very good results for the Phase 1/2 Trial of AXO-AAV-GM1 Gene Therapy for the Treatment of Infantile- and Juvenile-onset GM1 Gangliosidosis:

Phase 1/2 Trial of AXO-AAV-GM1 Gene Therapy for the Treatment of Infantile- and Juvenile-onset GM1 Gangliosidosis


C Tifft12 P D'Souza12 J M Johnston12 M T Acosta12 E R Nicoli12 C Rothermel12 A Thurm13 A Karunakara4 B Thorp4 P Ross4 J Jameson4 M Sheehan4 T Vaughn4 D Valencia4 E De Boever4 G Corcoran4

1: National Institutes of Health 2: National Human Genome Research Institute 3: National Institute of Mental Health 4: Sio Gene Therapies Inc.

Data is presented from an ongoing open-label, single-arm, dose-ranging Phase 1/2 trial (NCT03952637) of an investigational gene therapy AXO-AAV-GM1 (AAV9-GLB1) for treatment of GM1 gangliosidosis which is a rare, fatal, neurodegenerative lysosomal storage disorder. Subjects are administered AXO-AAV-GM1 intravenously following immune modulation with rituximab, sirolimus and glucocorticoids. Seven subjects have been enrolled in 2 cohorts: low-dose (1.5x1013 vg/kg), n?=?4 late-infantile-onset and n?=?1 juvenile-onset, follow-up 12-months; high-dose (4.5x1013 vg/kg), n?=?2 juvenile-onset, follow-up 6-months. AXO-AAV-GM1 was generally safe and well-tolerated. There were two serious adverse events unrelated to AXO-AAV-GM1. Six subjects had adverse events of AST or ALT elevations (≤5X above baseline) that did not require clinical intervention or have associated clinical sequelae and no adverse events of elevations in GGT or bilirubin were reported. At 6-months, CSF GM1 ganglioside was reduced to published normal levels and serum ß-gal enzyme activity increased above the lower limit of normal reference levels in the high-dose subjects. At 12-months, the CSF GM1 ganglioside remained below baseline levels in all low-dose subjects and serum ß-gal activity remained above baseline levels in two of five subjects. Volumetric MRI data in low-dose subjects showed maintenance of brain volume in four of five subjects at 12-months. There was no clinical evidence of disease progression in four of five low-dose subjects at 12-months or in the high-dose cohort at 6-months post-treatment as assessed by Vineland-3, Upright/Floor Mobility Score, Clinical Global Impression, and neurological examination. Further evaluation of these subjects is ongoing

Good luck and GOD bless,