OpRegen® is currently being tested in a Phase 1/2a clinical trial for the treatment of dry AMD with GA. OpRegen® is a cell-based product composed of retinal pigment epithelial (RPE) cells, derived from human embryonic stem cells (hESC) and administered as a cell suspension in ophthalmic Balanced Salt Solution Plus (BSS Plus).
Phase 1/2a Clinical Study Highlights from the 2020 Association For Research In Vision and Ophthalmology Annual Meeting:
- Significant update builds on prior reports of gains in visual acuity and provides a more comprehensive picture of treatment with OpRegen for dry-AMD
- Better visual acuity, reduced geographic atrophy (GA) progression and improved reading speed reported in first Cohort4 and first Orbit SDS patients
- Visit our Media page to watch the presentation on Phase 1/2a Clinical Trial Interim Results by Christopher D. Riemann, M.D., Cincinnati Eye Institute (CEI)
About the Phase 1/2a Clinical Study
This is a Phase 1/2a open-label, dose escalation safety and efficacy study of a single injection of human retinal pigment epithelium cells derived from an established pluripotent cell line and transplanted subretinally in patients with advanced dry AMD with geographic atrophy. The study will enroll approximately 24 patients, divided into 4 cohorts. The first 3 cohorts consisted solely of legally blind patients, with best corrected visual acuity (BCVA) of 20/200 or worse. The fourth cohort will include approximately 12 patients with vision ranging from 20/250 to as high as 20/64. Cohort 4 also includes patients treated with one of two formulations of OpRegen; the first 3 patients were treated with a formulation which required plating and preparation of cells one day prior to use. The remaining patients on Cohort 4 will be treated with an “off-the-shelf” or “thaw-and-inject” formulation of OpRegen which can be shipped directly to sites and used immediately upon thawing, which removes the complications and logistics of having to use a dose preparation facility. Until February 2020, staggered intervals within and between cohorts were applied to ensure patient safety and welfare. The primary objective of the Phase 1/2a study is to evaluate the safety and tolerability of OpRegen as assessed by the incidence and frequency of treatment emergent adverse events. Secondary objectives are to evaluate the preliminary efficacy of OpRegen treatment by assessing the changes in ophthalmological parameters measured by various methods of primary clinical relevance. Additionally, for the patients in Cohort 4 that receive subretinal delivery of OpRegen utilizing Gyroscope Therapeutics’ Orbit Subretinal Delivery System (Orbit SDS), objectives will include the evaluation of the safety of delivery of OpRegen using the Orbit SDS.