Our Work
At VisionBound Foundation, our work is centered on advancing science, empowering families, and accelerating progress toward a cure for Usher Syndrome Type 2A. We focus on four key areas that together create the greatest impact.
Gene Therapy Research
Targeting inherited retinal diseases
Clinical Trials Support
Funding & facilitating promising studies
Patient Advocacy
Empowering individuals & families affected by blindness
Global Partnerships
Collaborating with labs, universities, & biotech firms
Pioneering RNA Therapy for Usher Syndrome Type 2A
We are developing a new RNA therapy for Usher Syndrome Type 2A (USH2A) that aims to slow or stop the vision loss caused by changes in the USH2A gene. Our approach uses a technique called exon skipping, which works a bit like editing a sentence to skip over a word that doesn’t fit so the rest of the sentence still makes sense. In the case of USH2A, certain mutations disrupt the instructions for making usherin, a protein that is important for the health of the eye and ear. With exon skipping, we use small pieces of RNA, called antisense oligonucleotides (AONs), to guide the cell’s machinery to skip the faulty section of the gene’s message. This allows the body to produce a shorter but still working version of the usherin protein. By restoring some usherin function, our therapy has the potential to protect the light-sensing cells in the retina offering hope for people with Usher Syndrome Type 2A a chance to slow down or even stop the progression of their vision loss.
Our RNA therapy is being developed in accordance with the latest regulatory frameworks and guidance from the FDA, which provide standards on pharmacology, toxicology, chemistry manufacturing, and controls (CMC), as well as specific recommendations for individualized ASO therapies for rare diseases.