Non-invasive molecular tracking method that measures ocular drug distribution in non-human primates.

Intravitreal (IVT) injection has become the standard route for drug administration in retinal diseases. However, the ability to measure biodistribution of ocular therapeutics in large species remains limited, due to the invasive nature of some techniques or their lack of spatial information. The aim of this study was to develop in cynomolgus monkeys a non-invasive fluorescence imaging technology that enables tracking of IVT-dosed drugs and could be easily translated into humans.

Nonclinical Safety Evaluation of scAAV8- for Treatment of Retinitis Pigmentosa.

Retinitis pigmentosa is a form of retinal degeneration usually caused by genetic mutations affecting key functional proteins. We have previously demonstrated efficacy in a mouse model of deficiency with a self-complementary AAV8 vector carrying the gene for human under control of a short promoter (CPK850). In this article, we describe the nonclinical safety profile of this construct as well as updated efficacy data in the intended clinical formulation.