Report Summary

Single Dose 3-month Toxicity Study of Adeno-Associated Virus AAT Gene Vector in the C57/BL6 Mouse

Study Summary

Overall Study Design
The objective of this study was to determine the potential toxicity of adeno-associated virus expressing the alpha-one
antitrypsin gene rAAV2-CB-hAAT following a single injection into C57Bl6 mice The overall study was divided into two
separate parts the primary toxicology study and the biodistribution study See separate report for biodistribution study
The primary toxicology study consisted of 4 experimental arms The mice were injected intramuscularly with either PBS
vehicle control group or rAAV2-CB-hAAT at a total dose of 3 X 108iu 9 x 109vg  36 x 1011vgkg 1 X 1010iu 3
x 1011vg  12 x 1012vgkg or 26 X 1011 iu 8 x 1012vg  32 x 1014vgkg  Each arm consisted of 12 mice
which included 3 males and 3 females at each sacrifice time point which was day 21 or day 90 The primary safety
endpoint of this portion of the study was the histopathologic examination of the skeletal muscle lung heart liver lymph
nodes diaphragm brain spleen kidney pancreas jejunum and gonads The genomic DNA from these organs as well as
from blood was also assayed for the presence of vector genomes Clinical assessments were also measured by CBC and
serum chemistry analysis
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