Ocugen announced that dosing is complete in the second cohort of its phase 1/2 GARDian clinical trial for OCU410ST, a modifier gene therapy candidate being developed for Stargardt disease, as a 1-time treatment for life. The GARDian clinical trial will be conducted in 2 phases. Phase 1 is a multicenter, open-label, dose-ranging study consisting of 3 dose levels (low dose [3.75×1010 vg/mL], medium dose [7.5×1010 vg/mL], and high dose [2.25×1011 vg/mL]). Phase 2 is a randomized, outcome accessor-blinded, dose-expansion study, in which adult and pediatric patients will be randomized in a 1:1:1 ratio to either 1 of 2 OCU410ST dose groups or to an untreated group. Six patients have been dosed in the phase 1/2 clinical trial to date. An additional 3 patients will be dosed with the high dose (cohort 3) of OCU410ST in the dose-escalation phase.
“The completion of dosing for cohort 2 participants signifies an important clinical milestone for our pioneering modifier gene therapy,” Huma Qamar, MD, MPH, chief medical officer of Ocugen, said in a news release.