Potency Assay for AAV Vector Encoding Retinal Pigment Epithelial 65 Protein

This poster was presented at ARVO 2016 by Spark Therapeutics, Inc. Absorption Systems data was included.

Purpose

A validated potency assay for the release of medicinal products is required by the FDA prior to commercialization. SPK-RPE65 is a gene transfer vector being developed for the treatment of inherited retinal degeneration due to mutations in the retinal pigment epithelial 65 (RPE65) gene. In a Phase 3 trial, it demonstrated restoration of functional vision in subjects progressing toward blindness.
We developed a non-radioactive in vitro assay to measure the activity of SPK-RPE65, in anticipation of potential commercialization of this product.

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