Chair:

Kenneth Schafer, Greenfield Pathology Services Inc.

Co-Chair:

Helen Booler, Novartis Institutes for BioMedical Research, Switzerland

Primary Endorser: Ocular Toxicology Specialty Section


Endorser(s): Biotechnology Specialty Section


Endorser(s): Comparative Toxicology, Pathology, and Veterinary Specialty Section

The approval of the first ocular gene therapy in December 2017 marked a shift in the way we think about the treatment of ocular diseases, providing hope for patients with previously untreatable inherited retinal dystrophies. Its success has produced a relative explosion of interest in the field of ocular gene therapies. These therapeutic strategies, once considered solely as replacements of defective genes, have emerged as a potential method for transfecting cells in the eye by serving as endogenous therapeutic protein biofactories, which would result in the successful treatment of more prevalent diseases. As this is a new and rapidly evolving field, the goal of the session is to introduce the ocular gene therapy landscape, share current nonclinical study designs and development strategies implemented for ocular gene therapies, and consider current knowledge gaps and future directions. We will discuss common barriers to development, nonclinical safety findings, assessment of risk-benefit, and the translatability of nonclinical findings to patients. Experts will share experiences, case examples, and data from recent years. At the end of the session, there will be an extensive panel discussion to engage the audience on the presented topics. Attendees of this session will leave with an end-to-end overview of points to consider in the development of ocular gene therapies.