PHOTORECEPTOR CELLS FOR RETINAL AND MACULAR REPAIR

Smart overview of the Invention

Cell-based therapeutics are being developed for retinal and macular repair, focusing on specific retinal cell subtypes. These include rod photoreceptors and cone photoreceptors sensitive to short (S), medium (M), and long (L) wavelengths. The therapeutic approach aims to manufacture and utilize these defined cell types to address vision impairments caused by degenerative macular diseases.

Background on Degenerative Macular Diseases

Degenerative macular diseases, such as age-related macular degeneration (AMD) and Stargardt disease, lead to significant vision loss and are a primary cause of blindness. The degeneration of cone photoreceptor cells in the macula is particularly detrimental, resulting in the loss of high-acuity vision necessary for activities like reading and face recognition. Current treatment options primarily slow degeneration rather than repair damaged tissues, highlighting a critical need for effective therapeutic solutions.

Cell Composition and Ratios

The therapeutics involve specific cellular compositions with defined ratios of S, M, and L cone photoreceptors. For example, the ratio of L cones to M cones may range from 1.3:1 to 2.8:1, with variations for S cones as well. These ratios are designed to reflect natural distributions found in healthy human retinas, ensuring that the compositions closely mimic normal visual function.

Methods for Preparation and Application

Methods for preparing these cellular compositions include culturing organoids to achieve desired S-, M-, and L-cone ratios. Additionally, the use of engraftment techniques allows for the application of these cellular therapies directly into the macula of patients suffering from various forms of retinal degeneration. This targeted approach aims to restore function in areas impacted by diseases like AMD and other inherited conditions.

Regulatory and Research Support

The development of these therapeutic methods has received government backing through grants from the National Institutes of Health, indicating a recognized need for innovative treatments in ocular health. The research focuses on creating viable solutions that not only halt progression but also facilitate repair in degenerative retinal conditions.