Sio Gene Therapies Announces Positive Six-Month Follow-Up Data from Low-Dose Cohort of Phase 1/2 Trial of AXO-AAV-GM1 for GM1 Gangliosidosis
- Generally well-tolerated with a favorable safety profile in five patients
- Serum beta-galactosidase enzyme activity increased in all patients at all timepoints between Day 7 and Month 6, representing an approximate doubling in enzyme activity after gene transfer
- At Month 6, enzyme activity restored to 23-57% (mean: 38%) of normal reference levels
- All five children demonstrated signs of clinical disease stability as assessed by Vineland-3 Growth Scale Value, Upright and Floor Mobility, and Clinical Global Impression (CGI) scales
- High-dose cohort initiated in November 2020; two patients now dosed without complications
- Generally well-tolerated with a favorable safety profile in five patients
- Serum beta-galactosidase enzyme activity increased in all patients at all timepoints between Day 7 and Month 6, representing an approximate doubling in enzyme activity after gene transfer
- At Month 6, enzyme activity restored to 23-57% (mean: 38%) of normal reference levels
- All five children demonstrated signs of clinical disease stability as assessed by Vineland-3 Growth Scale Value, Upright and Floor Mobility, and Clinical Global Impression (CGI) scales
- High-dose cohort initiated in November 2020; two patients now dosed without complications