Translational Platform Program

Highlight Stats

Principal Agency or Investigator: Save Sight Now

Focus Area: Critical Research & Treatments

Grant Amount: $65,000

Grant Time: Three Years

Grant Details

Grant Overview

In partnership with Save Sight Now, The Fairfield Fund has launched the Usher 1B Translational Platform Program, a new research initiative designed to tackle two of the biggest obstacles standing between a promising therapy and a patient: understanding how that therapy actually behaves in human retinal cells, and figuring out how to safely deliver it into the retina in the first place. Rather than treating these as separate problems, the program brings them together in one shared research infrastructure, so scientists can study disease biology and test delivery strategies in the very same human-derived systems.

At the center of the program is a simple but powerful idea: build patient-derived human retinal models, then use those same models to test how different delivery approaches — including viral and non-viral methods — perform. This matters enormously for Usher 1B, because the gene responsible for the disease, MYO7A, is unusually large. Many of the delivery systems that work well for other genetic conditions simply can't carry a payload this size, which means delivery itself has become one of the central scientific bottlenecks in the field.

By pairing disease modeling with delivery testing, researchers can get earlier, more reliable answers about whether a given therapeutic approach is likely to work — before committing to the time and expense of animal studies or clinical trials. And because the resulting cell models and tools are stored long-term and made available to qualified researchers across the field, a single investment in this infrastructure can support many different research groups and many different therapeutic strategies for years to come.

Impact on Usher Syndrome 1B Patients

For families living with Usher 1B, the promise of gene therapy has always come with an asterisk: even if scientists design a treatment that could work, there has been no reliable way to test whether it can actually get to where it needs to go. This program changes that. By creating patient-derived retinal models alongside genetically corrected comparison cells, researchers can directly compare diseased and healthy tissue side by side, and by testing delivery strategies in those same human cells, they can start ruling in — or ruling out — approaches long before they reach a patient.

This is especially meaningful because MYO7A's size has forced the field to explore less conventional solutions, from dual AAV systems to non-viral platforms and other emerging technologies. Many of these approaches are still early and haven't been tested in models that truly reflect human retinal biology. This program gives them a proving ground.

Just as importantly, none of this work benefits only the Usher 1B community in isolation. The delivery challenges posed by MYO7A are a preview of what researchers working on other large-gene retinal diseases will eventually face. Lessons learned here — which delivery systems tolerate large payloads, how corrected cells compare to diseased ones, what early data actually predicts later success — will help de-risk translational research well beyond our own diagnosis.

Save Sight Now will track and report on this progress every year, both in terms of scientific infrastructure (how many cell lines and models have been built and validated) and ecosystem outcomes (how many researchers, partnerships, and new studies the platform has enabled). The goal isn't just to build one tool for one trial. It's to build lasting, shareable infrastructure that lets the field move faster, compare notes more easily, and bring safe, effective therapies to patients sooner — for our families, and for everyone facing the same delivery challenge behind them.