Atsena : Industry : Research : Usher Syndrome Coalition

Atsena Therapeutics

Research Continuum Graphic with the Preclinical section highlighted in yellow. The remaining sections are shown in grey. The order of the continuum is: Basic & Translational, Pre-clinical, Clinical trials: Phase I, Phase II, Phase III, Treatments-Phase IV

Dr. Shannon Boye and the team at Atsena are making rapid advancements in evaluating a dual AAV vector-based gene therapy to prevent blindness from MYO7A-associated Usher Syndrome (USH1B).

USH1B is caused by mutations in the MYO7A gene. USH1B patients are born profoundly deaf and begin to progressively lose vision in their first decade. The therapeutic gene to treat USH1B (MYO7A) is too large to fit inside a single AAV vector. To address this barrier, Atsena Therapeutics developed dual AAV vectors capable of delivering this large genetic payload, marking a potentially major advance in preventing blindness in patients affected by this devastating multisensory disorder.