DENVER — A gene therapy showed “encouraging visual function improvements” in patients with Usher syndrome type 1B, according to a phase 1/2 trial presented at the Association for Research in Vision and Ophthalmology meeting.
“Usher 1 syndrome is a disease in which children are born profoundly deaf,” Jayashree Sahni, MD, FRCOphth, PhD, EMBA, told Healio. “Over their lifetime, they start developing vestibular imbalance, and then slowly they have night blindness and progressive loss of vision.”
Participants received a low, medium or high dose of AAVB-081 (AAVantgarde Bio). The primary endpoint was