Report Summary

Toxicity Study of rAAV1 CB-hAAT (TFX) and rAAV1 CB hAAT (HSV) Administered Intramuscularly in the C57BL/6 Mice

Study Summary

Overall Study Design 
The Sponsor has previously conducted preclinical evaluations and a Phase 12 dose clinical study of rAAV1-CB-hAAT 
produced using a two-plasmid transient transfection method After completing the Phase 12 human clinical trial the 
Sponsor changed their test article manufacturing process to use a herpes simplex virus-based method to produce the 
vector This was done in order to scale up vector production and to achieve sufficient yields to administer higher doses 
The overall objective of this preclinical bridging study is to examine test articles produced by these two methods in parallel 
to determine 1 if any product contaminants resulting from the new HSV production method result in a different toxicity 
profile than occurs with the original TFX product and 2 to compare blood vector and human alpha-1 antitrypsin hAAT 
levels in animals as a function of product administered and as a function of HSV-produced vector dose 
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