Retinal Therapies Overview

Principles

Definition

Retinal therapies is a specialized field of biomedical research and clinical practice dedicated to the development and application of interventions aimed at treating diseases and disorders affecting the retina. This domain investigates a diverse array of therapeutic modalities, encompassing gene and RNA-based therapies, pharmacological agents such as anti-VEGF compounds and novel drug delivery systems, advanced biomaterials, and retinal prosthetics, all with the primary objective of preserving, restoring, or improving visual function by addressing the underlying retinal pathologies.

Ontological type

Core Modalities

Disease Targets

Delivery Strategies

Key developments

Key Figures

Biological Foundations era

D. A. Baylor [1] emerged as a leading figure at Harvard University [3] and the University of California, San Francisco [4] during the Biological Foundations era. This work, The photocurrent, noise and spectral sensitivity of rods of the monkey Macaca fascicularis [7], provided crucial functional benchmarks of primate rod physiology that helped frame retinal signal processing approaches relevant to therapies. Gerald A. Fishman [2] contributed during the era with appointments at Johns Hopkins University [5] and Columbia University [6]. His 1991 paper Rhodopsin mutations in autosomal dominant retinitis pigmentosa [8] helped illuminate the genetic degeneration mechanisms underlying photoreceptor loss and underscored the therapeutic potential of gene- and mutation-targeted strategies in retinal disease.

Molecular Signaling Therapies era

Jian‐xing Ma [1] is associated with Yale University [3] and Nanjing University [4] during the Molecular Signaling Therapies era. His key contribution was the demonstration that Rpe65 is necessary for production of 11-cis-vitamin A in the retinal visual cycle [7]. Edwin M. Stone [2] is linked to the Institute of Cancer Research [5] and the University of Pennsylvania [6] in this era. His notable finding that EFEMP1 mutations underlie both Malattia Leventinese and Doyne honeycomb retinal dystrophy [8] highlighted genetic targets for retinal disease therapies, shaping molecular approaches to diagnosis and intervention.

Precision Translational Therapies era

Chris A. Johnson [1] is affiliated with the University of California, San Diego [3] and the University of California, Los Angeles [4] in this Precision Translational Therapies era. His key contribution in this era was the 2017 randomized, controlled, open-label phase 3 trial of voretigene neparvovec (AAV2-hRPE65v2) [7] for RPE65-mediated inherited retinal dystrophy, which demonstrated efficacy and safety and helped establish genotype-guided gene therapies as a clinical reality. Edwin M. Stone [2] has been affiliated with The University of Texas Southwestern Medical Center [5] and Johns Hopkins University [6] during this era, and the same phase 3 trial [7] features prominently in his contributions. Edwin M. Stone [2] helped translate these findings into translational pipelines by shaping regulatory and patient-selection frameworks for precision retinal therapies, as exemplified by the phase 3 trial [7].