Cell and Gene Therapy for Retinal Dystrophies: Models, Delivery, and Imaging in Preclinical Research

Hosted by the SOT Ocular Toxicology Specialty Section

Retinal dystrophies, or inherited retinal diseases (IRDs), are a diverse group of disorders marked by progressive loss of photoreceptors or retinal pigment epithelium, leading to retinal atrophy and blindness. Common IRDs include retinitis pigmentosa, Stargardt disease, choroideremia, and Leber congenital amaurosis. Recent research and various clinical trials indicate that cell and gene therapies hold promise as potential treatments for IRDs to recover loss of function caused by mutations in retinal and RPE layers. Before receiving US FDA approval for clinical use, these therapies must undergo rigorous evaluations for safety and toxicity potential and dose optimizations in animal models. In this webinar, we will cover an overview of cell- and gene-therapy approaches for IRDs; various types of cell and gene therapies; criteria for selection of appropriate animal models; delivery routes for targeting various ocular compartments; the role of non-invasive in vivo imaging modalities in preclinical studies; study design considerations based on regulatory guidelines; and illustrative case presentations demonstrating key preclinical strategies.

Speaker:

Sandeep Kumar, PhD, Director of Ophthalmology, Pharmaron