Sio Gene Therapies — Technical Presentation (May 2021)
4 slides from Sio Gene Therapies's technical presentation, May 13, 2021
Slide 1: Preliminary Results from the AXO-AAV-GM1 (AAV9-GLB1) Clinical Trial for GM1 Gangliosidosis
Preliminary Results from the AXO-AAV-GM1 (AAV9-GLB1) Clinical Trial for the Treatment of GM1 Gangliosidosis Cynthia Tifft MD, PhD National Human Genome Research Institute, NIH Exhibit 99.2 1
Slide 2: Forward Looking Statements and Disclaimer
3 Forward Looking Statements and Disclaimer This presentation is for informational purposes only and not for any other purpose. It may include forward-looking statements about Sio Gene Therapies Inc. and its clinical, pre- clinical, regulatory and manufacturing activities. Such statements are based
Slide 3: Biomarkers of Disease Progression or Stabilization
11 Biomarkers of Disease Progression or Stabilization 0 50 100 150 200 250 0 90 180 GM1 Ganglioside in CSF G M 1 G an gl io si de (n g/ m L) Pt1: Juvenile; Pt2-5: Late infantile LLN= Lower limit of normal from 30 serum samples of 30 presumed healthy adults 1. Izumi T et al. Ped Neuro 1993;9:297-300,
Slide 4: AXO-AAV-GM1 Intravenous, Low-dose Summary
18 AXO-AAV-GM1 Intravenous, Low-dose Summary at 6 Months AXO-AAV-GM1 was generally well-tolerated with a favorable safety profile ß-gal enzyme activity in serum increased from baseline by 33%-128% across 5 subjects GM1 ganglioside in CSF decreased from baseline by 18-49% across 4 subjects demo
Sio Gene Therapies — Technical Presentation (May 2021) on 6ix