Annual Newsletter - 2024

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Dear Friends,

As the holiday season draws near, I find myself reflecting on the incredible journey we’ve been on at the Usher III Initiative—a journey driven by determination, discovery, and hope. For those who have been with us since the beginning, thank you. Your belief in our mission has carried us through every milestone. For those who are new to our story, welcome. You’ve joined us at an extraordinary moment in our quest to bring sight and sound back to those of us living with Usher Syndrome Type 3.

Our story didn’t begin with my diagnosis of Usher III; it began with a lesson my father taught me: that even in the face of overwhelming challenges, we have the power to create change. That belief became the foundation of the Usher III Initiative, a mission to take on what many thought was impossible. Today, that mission is closer to becoming a reality than ever before.

A Journey Fueled by Hope

Usher Syndrome Type 3 is a progressive condition, stripping individuals of their ability to see and hear and profoundly altering their lives. It’s a diagnosis that historically came with little hope. But my father, Dick Elden, taught me NOT to accept that tragic narrative.

Our first milestone occurred when we discovered the root cause of the disease: a defect in a protein called Clarin-1. Preserving the integrity of this protein could prevent the loss of hearing and vision. This discovery led us to high-throughput screening of thousands of compounds, searching for one that could stop the progression of this disease.

Then came our second big win: we found a molecule with the potential to change everything. Manufacturing the compound, ensuring its safety, and scaling up production were monumental tasks. But with the help of brilliant researchers, devoted partners, and generous supporters like you, we successfully completed this phase of the process.

A Transformative Milestone

Today, I am thrilled to share that our Phase 1 clinical trials are officially underway in Australia.

This milestone represents the culmination of years of dedication, collaboration, and an unwavering belief in the inevitability of a cure. It is a moment of triumph—not just for us, but for the many individuals and families whose lives are impacted by Usher Syndrome Type 3.

But there’s more: as our Phase 1 trials progress, we are also conducting a natural history study in partnership with the Foundation Fighting Blindness to better understand the progression of this condition. This work will guide our next steps and ensure that we move forward with precision and purpose.

Defying the Odds

Every milestone we’ve surpassed has been against the odds. Out of 7,500 compounds identified in drug discovery, only five make it to clinical trials. We are one of those special five. And now we stand on the threshold of a new chapter, one that could transform lives for generations to come.

Some said it was impossible. Some thought we were reaching too far. But here we are, successfully moving forward from milestone to milestone.

Crossing the Finish Line

While we are closer than ever, we have not yet crossed the finish line. Once Phase 1 is complete, we will submit our compound to the FDA for approval and prepare to launch Phase 2 trials in Usher III patients. This will require significant additional funding, and we will continue to seek new strategic partnerships to complement our efforts.

The Power of Your Support

This holiday season, as I reflect on how far we’ve come, I am overwhelmed with gratitude—for the researchers who have worked tirelessly, for the partners who have stood by us, and for you—our supporters—who believed in our vision even when it seemed like a distant dream.

As we move toward Phase 2—the next big step—I ask you to continue believing in our mission. I would be deeply grateful if you would consider celebrating our recent milestones by making a generous gift to empower us to tackle Phase 2, and begin to treat patients with Usher III.

Together, we’re building a future filled with sight and sound—a future where Usher Syndrome Type 3 no longer defines lives but inspires breakthroughs.

Thank you for being a part of this extraordinary story.

Wishing you a holiday season filled with hope, joy, and the knowledge that you are making a very real difference.

With heartfelt gratitude,

Cindy Elden

President & Co-Founder, Usher III Initiative