Stephen Simone is the father of Francesco and Vincenzo, two young boys born with Usher syndrome. The condition leaves children deaf from birth, without a sense of balance, and facing progressive sight loss that typically sets in around primary school age. When the diagnosis came, Stephen’s response was immediate: do something.
In October 2024, he founded the Silent Sight Foundation with a singular mission — to find treatments and cures for the blindness caused by Usher syndrome. Within a year, the Foundation had built real fundraising momentum. But with funds came a critical question: where should they go?
“Obviously when you’ve gone out to the public and asked for their support, you want to make sure that you’re utilising those funds and resources diligently,” Stephen explains. Coming from outside both the not-for-profit and medical research sectors, he knew the stakes were too high to navigate alone. The Foundation reached out to Research Strategies Australia.
As a family-led charity with no prior experience in either the not-for-profit sector or the highly regulated world of medical research, Silent Sight Foundation faced a daunting landscape. The path from diagnosis to treatment involves complex scientific, regulatory, and commercial systems and a wrong turn could mean years of delay for children already racing against the clock.
Stephen came to RSA with a clear working theory: fund Usher syndrome research directly. It seemed like the obvious path. But was it the most effective one? And were faster, smarter routes being overlooked?
• A clear, evidence-based strategic direction informed by the global research and treatment landscape
• Confidence that its resources would be deployed where they could have the greatest impact for Australian children
• The credibility to open doors with leading researchers, clinicians, regulators and major donors
“Our focus was: Our boys have this issue. We just want to fix it. We need to fund research. That was our lens coming into it.” - Stephen Simone, Founder, Silent Sight Foundation
RSA took Silent Sight Foundation through a comprehensive strategic consultation process — beginning not by validating assumptions, but by challenging them. The first move was to zoom out.
Rather than confining the engagement to a single funding pathway, RSA mapped the broader ecosystem of Usher syndrome research, treatment development, and patient access. Nearly 40 global experts were engaged across more than 25 sessions — including patient advocacy leaders from Australia, Ireland, Europe and the United States; biotechnology companies and contract research organisations; academic researchers from Johns Hopkins University, the University of Melbourne, and Murdoch University; and government, regulatory, and clinical trial specialists.
This process surfaced pathways the Foundation had not previously considered: N-of-1 and small cohort clinical trial models that could dramatically reduce the cost and complexity of running trials; not-for-profit biotech structures that bypassed commercial barriers slowing treatment development; and lessons from other rare disease communities that had already successfully navigated the same challenges.
• Deep system understanding:
Rather than accepting the Foundation’s initial framing, RSA brought the expertise to map the full treatment development ecosystem — helping SSF understand where it could have the highest impact relative to its size and resources.
• Engaging stakeholders:
RSA’s existing networks in the rare disease space opened doors and gave the Foundation immediate credibility with the calibre of experts it needed to inform its strategy.
• Iterative testing:
Priorities were stress-tested in a full-day strategic workshop, mapping ambitions across four dimensions — complexity, cost, timelines and impact — to identify where SSF should focus first.
• Actionable roadmap:
The process produced not just a set of priorities, but a detailed 10-year implementation roadmap with measurable milestones, task-level detail and clear timelines.
“I was actually expecting something a little bit more high level. To have that detail was just incredible.” - Stephen Simone, Founder, Silent Sight Foundation
The engagement produced a complete shift in strategic direction — and immediate, tangible results.
Rather than directing all resources into a single research stream, Silent Sight Foundation now operates across two strategic layers: building the ecosystem infrastructure that allows treatments to move faster and selectively accelerating direct translational programs for children with Usher Syndrome.
The RSA-developed roadmap has been put to immediate use in major donor fundraising conversations, generating strong momentum. “We’ve got some exciting stuff happening with that, which I’m really looking forward to,” Stephen notes.
Several of the most promising opportunities to emerge — including N-of-1 trial models and not-for-profit biotech partnerships — came directly from the stakeholder consultation process. “Some of the unexpected opportunities that came out of the stakeholder sessions are looking to be the ones we’re going to be really diving into,” Stephen reflects.
Having RSA in their corner also gave Silent Sight Foundation the legitimacy to attract and engage the calibre of experts that a family-led charity might otherwise have struggled to access. “I think they saw that we were serious about achieving our goals, and I think they got excited by that too,” says Stephen.
“We’re not here to feel good, we’re here to do good — and that’s why we engaged RSA. That engagement gave us legitimacy with some of those key stakeholders, and I think they saw that.” - Stephen Simone, Founder, Silent Sight Foundation
Silent Sight Foundation now has what it came for: answers and a direction. But it also has something it didn’t expect — a strategy that has fundamentally reframed what the Foundation is and what it can achieve.
By zooming out from a single assumption to the full landscape of possibility, RSA helped Silent Sight Foundation understand not just where to direct its resources, but how to position itself as a credible, strategic actor in a complex global ecosystem — one that is genuinely capable of accelerating the arrival of treatments for Australian children with Usher syndrome.
For a family-led foundation where the consequences of every strategic decision are felt by two children — Francesco and Vincenzo — and by every other family living with Usher syndrome in Australia, that clarity is everything.
“The stakes are so high. You wouldn’t want to make those decisions without the right sort of advice behind you — and RSA were able to provide that advice and guidance that we needed.” — Stephen Simone, Founder, Silent Sight Foundation
What the engagement also revealed was just how complex the global Usher Syndrome space truly is. Dozens of organisations — advocacy groups, research foundations, biotechs and clinicians — are working toward overlapping goals from different angles, in different countries, often without full awareness of each other. One of the unexpected gifts of the RSA process was learning how to navigate that landscape: where to collaborate, where to lead, and where to let others carry the load.
The opportunity, Stephen believes, extends well beyond Silent Sight Foundation. It is a model for how the entire rare disease community could move faster — if it had the right strategic guidance.
“To be honest, if I had it my way, I would actually engage RSA to build out the global strategy to treat and kill the blindness caused by Usher syndrome.” - Stephen Simone, Founder, Silent Sight Foundation
We acknowledge the Dharawal people as the Traditional Owners of this land and pay our respects to Elders past and present. This is, was, and always will be Aboriginal land.
