Disposable Instrument for Broader Patient Access to Vision Restoration Therapy

DESCRIPTION (provided by applicant): Retinal diseases such as age-related macular degeneration (AMD), retinal dystrophies, and retinitis pigmentosa (RP) affect millions of Americans. As the population ages, this number will grow significantly. Many people will lose their sight due to degeneration of photoreceptors and/or retinal pigment epithelium (RPE). The majority of clinical studies performed to date have focused on therapies to address the early stages of these diseases in attempts to rescue the photoreceptors, the light-sensitive cells of the retina. However, once the photoreceptors have died, they cannot be regenerated. Many patients with advanced retinal diseases not only need new photoreceptor cells and new RPE but other cells of the retina as well. Development of an FDA-approved therapy to transplant these cells to patients with retinal degenerative diseases is a compelling research opportunity. Much work has been done in rat models and human patients to prove that degenerated photoreceptor and RPE cells can be replaced with new cells and connect with the remaining inner retina to restore eye sight. In this protocol, sheets of freshly harvested RPE and immature retina (containing cells that will develop into light- sensitive photoreceptors) are transplanted into the back of the rat or human eye under the retina. The transplants develop into different cell types and integrate with the damaged host retina. To make this protocol widely available, the objective of this research is to prove feasibility of a disposable, low-cost surgical instrument that can provide the precision needed for placement of the delicate RPE/retina tissue sheets during surgery. The instrument design must also be suitable for high-volume, low-cost production in a sterile one-way package. The specific aims are to: 1) design the components (hand piece, mandrel, and nozzle) of a fully integrated, production-ready disposable surgical instrument that will meet FDA approval requirements for medical devices; 2) fabricate three engineering prototypes and test these in eye bank (cadaver) eyes to demonstrate instrument performance; and 3) develop standardized production processes to enable low-cost manufacture and sterile packaging. Success in the proposed research will lead to development of a disposable instrument that will allow broader patient access to retinal transplantation as a vision restoration therapy. PUBLIC HEALTH RELEVANCE: Millions of Americans suffer from degenerative diseases of the retina and lose their sight. By transplanting sheets of retinal tissue, vision can be saved and improved. The proposed research will develop a special surgical instrument that will help make transplant of retinal tissue broadly available as a therapy.