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Report Summary

Assessment of the Safety and Biodistribution of rAAV6-BARKct Administered Once in Male and Female Sprague Dawley Rats


Study Summary

Overall Study Design 
The objective of this study was to evaluate the potential for off-target toxicity of multiple dose 
levels of AAV6-Beta ARKct following systemic intravenous administration in rats Intravenous 
administration provided a worst case scenario for vector and transgene product leakage from the 
heart following retrograde coronary vein delivery the intended route of administration in the 
proposed clinical trial 
The sponsor has previously demonstrated that rats are biologically responsive to human BetaARKct transgene 
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