Report Summary

Preclinical Study of Adeno-Associated Virus Mediated Gene Therapy for Retinal Dystrophy (AAV2-VMD2-hMerTK)

Study Summary

Overall Study Design 
The objective of this study was to determine the potential toxicity and biodistribution of adeno-associated virus serotype 2 
expressing the MERTK tyrosine kinase receptor protein from the VMD promoter AAV2-VMD2-hMerTK following subretinal 
injection into Sprague Dawley rats 
The study consisted of 3 experimental groups The rats received a single unilateral subretinal injection with either BSS 
vehicle control group or AAV2-VMD2-hMerTK at a total dose of 4x10e8 or 4x10e9 vg The control arm consisted of 4 rats 
which included 2 males and 2 females and sacrificed at a time point of 90 days -3 days The low and high dose test 
article arms consisted of 10 rats which included 5 males and 5 females and sacrificed at a time point of 90 days -3 days 
The primary safety endpoint of this study was the histopathologic examination of target and peripheral tissues and detection 
of vector genomes in the skeletal muscle quadriceps diaphragm heart brain lung spleen liver kidney pancreas 
gonads jejunum parotid gland mesenteric lymph node Injected and contra lateral eyes with optic nerve Clinical 
assessments were also measured by CBC and serum chemistry analysis 
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