Built to Accelerate a Treatment for USH1B
Save Sight Now is more than a just a funding organization. We are a patient-driven research engine built to identify the critical barriers and highest-potential opportunities that can accelerate progress toward a treatment for Usher syndrome.
Building on progress
Before becoming an independent nonprofit, Save Sight Now had already raised more than $3 million in partnership with the Foundation Fighting Blindness, built and funded a portfolio of scientific programs spanning critical assays, disease models, patient-derived cells, and natural-history studies, and established one of the field’s broadest USH1B research networks.
$3M Raised
In partnership with the Foundation Fighting Blindness before becoming an independent nonprofit.
7 Research Programs Funded
Across assays, disease models, patient iPSC’s, and natural-history studies.
Deep Expert Network
Specialists in retinal biology, gene editing, large-gene delivery, small-molecule development, manufacturing, and regulatory strategy.
“Save Sight Now has been visionary in its pursuit of therapies for children with USH1B — thoughtful and courageous in directing limited resources toward paths most likely to yield sight-saving treatments. As a researcher in inherited retinal disease, I find it deeply inspiring that one family has done so much to rally support, raise funds, and advance the most promising paths to a cure.”
- Martha Neuringer, PhD Professor, Oregon National Primate Research Center
Shaping the Roadmap
01 | Map the Landscape
In December 2025 We convened ~30 experts in North Carolina to review the USH1B landscape, critical gaps, and barriers to treatment.
02 | Set the Priorities
At ARVO 2026, experts reconvened at our USH1B Workshop to pressure-test discovered opportunities and refine where SSN could have the greatest impact.
03 | Build the Programs
Today, priorities are becoming defined research programs with clear experiments, partners, and critical decision points.
The strategic focus of a biotech. The urgency of a patient-led organization.
Our model combines rigorous development strategy, specialized expertise, and patient-led urgency to focus resources where they can have the greatest impact.
Holistic Development Strategy
We evaluate the full path to treatment—not just the science—from therapeutic approach and delivery through safety, manufacturing, regulatory strategy, and clinical feasibility.
Deep Expert Network
We draw on an embedded research team through the Brydge Solutions Initiative at Odylia Therapeutics, our Scientific Advisory Board, and a broad specialist network with deep experience in inherited retinal disease and therapeutic development.
Focused, Patient-Led Execution
We pair scientific rigor with relentless urgency and accountability, keeping resources focused on the work most likely to move treatments toward patients.
Move with urgency. Pursue multiple paths. Scale with evidence.
These principles guide how we prioritize research across a portfolio of parallel programs—favoring approaches that can reach patients sooner, creating multiple credible paths to treatment, and committing additional capital only as the evidence strengthens. Parallel development protects time. Disciplined stage gates protect capital.
Speed to Treatment
Prioritize work that can materially shorten the path to patients or preserve the therapeutic window.
Multiple Shots on Goal
Advance multiple credible therapeutic approaches in parallel rather than depend on a single path to treatment.
Evidence-Gated Funding
Identify the gating question, fund only the work needed to answer it, then advance, pivot, or stop based on the results.
Where We’re Investing Today
These are our most developed near-term funding priorities — not the limits of our work. Our objective is to bring a USH1B therapy into clinical trial by 2030.
01 | Non-Viral Large-Gene Delivery
Developing new ways to deliver full-length MYO7A to retinal cells, creating options beyond dual AAV and supporting multiple therapeutic programs.
02 | Base & Prime Editing
Developing mutation-specific editing approaches to directly correct eligible MYO7A variants and create repeatable paths for additional patients.
03 | Small-Molecule Drug Repurposing
Screening existing drugs and compounds for their ability to protect photoreceptors and slow vision loss while longer-term therapies advance.
04 | Enabling Science & Tools
Building patient-derived cells, assays, disease models, and other tools needed to test therapies and determine which programs should move forward.
Help Move the Strongest Paths Forward
With promising new technologies and a focused strategy to accelerate research, a treatment for USH1B is closer than ever. How many programs we can fund — and how quickly we can advance them — depends on the funding we can raise.