Eye on the Research

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A list of gene replacement studies currently underway that may potentially benefit patients with Usher syndrome. Please refer to the link under each trial for more information on that trial. Please be aware, all information provided are for informational purposes only, and are in no way endorsed or approved by Usher Syndrome Ireland. 

Pre-Clinical Development Stage

• Atsena Therapeutics is making rapid advancements in evaluating a dual-AAV vector-based gene therapy that could potentially prevent blindness from MYO7A-associated Usher Syndrome 1B (USH1B). More information can be found here: https://atsenatx.com/programs/ush1b/ 

Early Research Stage

• Standard USH2A genes are too large to fit into a single AAV used in gene therapy to replace a faulty gene with a functional copy. Iveric Bio is currently in early research stage aiming to develop a smaller USH2A construct (minigene) to fit into an AAV and transcribe a functional protein. More information can be found here: https://ivericbio.com/why-iveric/