Report Summary

Toxicology and Biodistribution Study of rAAV-AAT Vectors in Rabbit Tissues

Study Summary

Overall Study Design 
The objective of the animal portion of this study was to determine the biodistribution of a rAAV2 vector rAAV2-CB-hAAT 1 
X 1013 vgkg in skeletal muscle blood semen and gonads of New Zealand White Rabbits Furthermore excipient salt 
concentration 350 or 650mM and injection volume 03ml or 30ml effects on vector genome biodistribution were 
evaluated An additional arm of rabbits was injected with UF11 rAAV2-CB-GFP as a procedural control at the same dose 
1 X 1013 vgkg at 350mM salt and 30ml injection volume 
The objectives of the study performed under Good Laboratory Practices covered by the issued study protocol 01UF02rAAT 
were to evaluate serum chemistries and blood PCR parameters at the following time points baseline 6hr 18hr 1 day 3 
days 7 days 2 weeks and 4 weeks sacrifice date Total semen and the motile fraction of semen were analyzed by 
real-time PCR at baseline day 1 day 7 and 4 weeks post injection for the presence of vector genomes Skeletal muscle 
and gonads were assayed at necropsy by real-time PCR for presence of vector genomes 
The objectives for histopathological analysis were to evaluate the skeletal muscle gonads kidney heart liver lung 
pancreas and spleen for signs of inflammation or other abnormalities that may have resulted from the test article injection 
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