First Ever Human Clinical Trials: A Step Toward Hope
To Our Friends & Supporters,
As you know, we have been working hard for 25 years to make a treatment for Usher III patients a reality. That is why I’m thrilled to share the groundbreaking news regarding not one but two exciting developments that will bring us closer than ever to life-changing treatments for those living with this rare and debilitating condition.
First Ever Human Clinical Trials: A Step Toward Hope
Beginning next week, we launch the next phase of our ambitious journey: our first-in-class human clinical trial of our novel compound, BF844.
In brief, BF844 is small molecule developed by the Usher III Initiative to work as a “chaperone” designed to target the mutant CLRN1 protein in the N48k gene (which is the root cause of the Usher III condition).
For the first time in history we will be conducting the human clinical trial on BF844, which will last 15 months and prove instrumental in evaluating the safety of this ground-breaking treatment for humans.
Beyond the science, this trial represents a new day of hope for the countless individuals and families affected by Usher III, which as you know currently has no cure. We are incredibly excited about what this could mean for the future of patients living with USH3A, and it is your support that is making this all possible!
Mapping the Future: A Study to Shape Treatments
In addition to our new clinical trial, we are also proud to announce our collaboration with The Foundation Fighting Blindness to conduct a Natural History Study to chart the progression of vision and hearing loss in USH3 patients over time.
By closely monitoring how the disease advances clinically in patients, we will unlock valuable insights that will guide future treatment strategies and interventions. The information we gather in collaboration with the Foundation Fighting Blindness will help us identify the optimal time to intervene, potentially altering the course of this disease for future generations.
We Need Your Support to Make These Dreams a Reality
These two initiatives represent a giant leap forward in our mission, but they are just the beginning. To make this hope a reality for families facing the uncertainty of Usher III, we need your help.
Every donation brings us one step closer to a world where Usher III is not a life sentence, but a condition that can be treated, managed, and ultimately cured. Your generosity means the world to families who have waited for so long, and now finally see hope on the horizon.
Together, we can give them what they have dreamed of for years—a future filled with light, sound, and possibility.
Thank you for standing with us in this fight. Your unwavering dedication has brought us this far, and with your continued support, we will bring hope to life.
Or, if you prefer, you can make checks payable to Usher III Initiative and mail to Michelle Pell at:
Usher III Initiative
191 N. Wacker Dr., Suite 1790
Chicago, IL 60606
With deepest gratitude,
