Early “optimistic” data are available for the first two patients receiving Lineage Cell Therapeutics’ thaw-and-inject formulation of OpRegen via a new subretinal delivery system, according to a press release.
OpRegen is under study as a retinal epithelium transplant therapy in patients with advanced dry age-related macular degeneration with geographic atrophy.
The phase 1/2a study aims to evaluate the safety and efficacy of a single injection of human retinal pigment epithelium cells derived from an established pluripotent cell line in 24 patients divided into four cohorts. The first three cohorts included legally blind patients with best corrected visual acuity of 20/200 or worse; the fourth cohort will ultimately include approximately 12 patients with better baseline vision, ranging to 20/64.
“We are increasingly optimistic about the data we are collecting in dry AMD,” Lineage Cell Therapeutics CEO Brian M. Culley, said in the release. “We have treated five patients in cohort 4, those with less advanced disease, which more closely match our intended patient population. At the longest available assessment point for each patient, all five have shown an increase in the number of letters they can read on an ETDRS eye chart. Importantly, these gains have been maintained for as long as 15 months, which is the longest time point for which we have collected data in the better vision cohort.”
Two of the five patients in cohort 4 have received the thaw-and-inject formulation via a new subretinal delivery system by Gyroscope Therapeutics. At 6 months after treatment, one patient gained five lines of vision, improving from 20/250 at baseline to 20/100 in the treated eye. A similarly dosed second patient has shown small improvement in visual acuity 14 days after treatment. No unexpected complications were noted in either patient.