Gene Therapy for Usher Syndrome Type 1B (MYO7A Mutations): Oxford Biomedica

Description: Oxford BioMedica, a gene therapy company in the U.K., has launched a Phase I/IIa clinical trial of its UshStat® gene therapy for people with Usher syndrome type 1B. The trial has received regulatory authorization and orphan product designations in the U.S. and Europe. The company is starting the trial at Oregon Health & Science University (Portland, Oregon). As many as 18 participants will be enrolled in the two-year study. Three dose levels of the treatment will be evaluated for safety, tolerability, biological activity, and changes in visual function.

Trial Status:


FDA Approves Start of Usher Syndrome Gene Therapy Clinical Trial

Partial List of Participation Criteria:


  1. Diagnosis of Usher syndrome type 1B caused by MYO7A mutations
  2. 18 years and older


Clinical Contacts:

Richard Weleber, M.D.
Phone: (503) 494-3795
Casey Eye Institute, Oregon Health & Science University
Portland, Oregon 97239

Additional Information:

UshStat clinical trial listing on