Yale Startups in Formation: Challenges & Opportunities in Retinal Gene Therapy

With approximately 1.3 billion people living with some form of vision impairment, sight-restoring therapy for the visually impaired and blind is a major unmet medical need. Ocular gene therapy is a rational choice for restoring vision or preventing the loss of vision because most blinding diseases originate in cellular components of the eye, a compartment that is optimally suited for the delivery of genes. The eye particularly well-suited for diagnostic and therapeutic exploration: easy access, small volume, high internal compartmentalization, and stable cell populations. The optical transparency of the eye allows direct visualization with high-resolution imaging and precise evaluation of disease stage and response to therapy. Moreover, the relative immune privilege of the eye, especially the subretinal space, reduces adverse responses to injected vectors and gene products and many of these diseases have a genetic origin or genetic component.

In this session, Dr. Sahel will provide an overview of the field, describe major advances in the field of ocular gene therapy, and discuss proof-of-concept studies which are under way to evaluate the safety and efficacy of human gene therapies, with special focus on his own work in neuroprotection, optogenetics and clinical trials. Dima Kuzmin will discuss the venture potential in this promising space.

Host:
James Boyle, Executive Director, Yale OCR

Chat Moderator:
Michelle McQueen, Communications, Yale OCR

Session Moderator:
Roy Herbst, Professor, Yale School of Medicine

Featured Speaker:
Jose-Alan Sahel, Professor, University of Pittsburgh

Panelist:
Dima Kuzmin, Founding Partner, Adjunct Professor, 4Bio Capital/Yale