EyeCRO Contributes to LUXTURNA® Potency Assay Validation
We’re proud to share that EyeCRO CEO Dr. Rafal Farjo is a co-author on a new publication validating a cell-based potency assay for LUXTURNA®, the first gene therapy approved in the U.S. for an inherited retinal disease.
As stated in the publication:
“This represents the first report of validation studies supporting an in vitro cell-based relative potency assay for an AAV vector, which was used to evaluate lot-to-lot consistency, stability, and comparability following manufacturing changes and to successfully launch Luxturna, the first gene therapy approved in the US for a genetic disease.”
The validated assay measures LUXTURNA’s biological activity by quantifying RPE65 gene expression in transduced retinal pigment epithelial cells—a critical step in ensuring that each manufactured batch meets defined potency and quality standards.
This work not only supported the successful commercial launch of LUXTURNA, but also provides a strong example of how in vitro potency assays can be developed and validated to meet regulatory expectations for gene therapy products.
Through Dr. Farjo’s contribution, EyeCRO is proud to support the advancement of science and quality standards in gene therapy development.
