Ocular gene therapies

At Experimentica, we offer specialized in vitro, in vivo, and downstream analytical services to support the development of gene therapies targeting ocular diseases. From early in vitro studies to in vivo biodistribution and efficacy, we provide end-to-end expertise designed to advance your programs at our AAALAC-accredited and BSL-2 laboratories.

Comprehensive capabilities for ocular gene therapy

Our team routinely performs in vitro transduction, expression, and cell viability studies to confirm early safety and expression of the sponsors’ vectors in specialized cell types (hiPSC-RPE, HEK293…) before moving into in vivo models.

Experimentica can design customized in vivo studies to evaluate the biodistribution and efficacy of a variety of vectors (AAVs, lentiviruses, HSV-1, exosomes…) in pup and adult mice, rats, and rabbits.

A key aspect of our expertise relies on the route of administration, specifically preferred injection routes for ocular gene therapies such as subretinal, intravitreal, and suprachoroidal routes, coupled with imaging to confirm successful injections when relevant.

Our scientists have a track record of testing gene therapies in models of wet AMD, dry AMD and Geographic Atrophy, Glaucoma, and Retinal Degeneration and Retinitis Pigmentosa models (rd1, rd10, Rho^P23H…).

As part of the tissue and molecular analysis, we offer routine or custom histology and immunohistochemistry services as well as gene expression, vector biodistribution, and protein quantification assessed through qPCR, Western blot, and ELISA, giving sponsors detailed insight into the performance and safety of their gene therapy drug candidate.

Representative images of retinal cross-sections immunostained against GFP (magenta) and counterstained with DAPI (cyan)

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