RG6501 (OpRegen) Phase 1/2a Clinical Study 24 Month Results to Be Featured at 2024 Retinal Cell & Gene Therapy Innovation Summit - Seite 2

About the Foundation Fighting Blindness

The Foundation Fighting Blindness was established in 1971 by a passionate group of families driven to find treatments and cures for inherited retinal diseases that were affecting their loved ones. At that time, little was known about these blinding retinal diseases. Very little research was being done, and there were no clinical trials for potential treatments. Today, the Foundation Fighting Blindness is the world’s leading private funder of retinal disease research. That funding has been a driving force behind the progress toward cures, including the identification of more than 270 genes linked to retinal disease, and the launch of over 40 clinical trials for potential treatments. For more information, visit: https://www.fightingblindness.org/ or follow them on X/Twitter @FightBlindness.

About OHSU Casey Eye Institute

OHSU Casey Eye Institute is a premier academic medical center providing eye care for adults and children in the Pacific Northwest and beyond. Since 1945, OHSU Casey Eye Institute doctors and vision scientists have worked side by side to discover the causes of eye disease and find new treatments. For more information, visit: https://www.ohsu.edu/casey-eye-institute or follow them on X/Twitter: @CaseyEye.

About the OpRegen Phase 1/2a Study

The Phase 1/2a study is an open-label, single-arm, multi-center, dose-escalation trial evaluating a single administration of OpRegen delivered subretinally in patients with bilateral GA secondary to AMD. Twenty-four patients were enrolled into 4 cohorts. The first 3 cohorts enrolled only legally blind patients with a best corrected visual acuity (BCVA) of 20/200 or worse. The fourth cohort enrolled 12 patients with impaired vision (BCVA from 20/65 to 20/250 with smaller mean areas of GA). Cohort 4 also included patients treated with a new “thaw-and-inject” formulation of OpRegen, which can be shipped directly to sites and used immediately upon thawing, removing the complications and logistics of having to use a dose preparation facility. The primary objective of the study was to evaluate the safety and tolerability of OpRegen as assessed by the incidence and frequency of treatment-emergent adverse events. Secondary objectives include evaluating the preliminary activity of OpRegen treatment by assessing the changes in ophthalmological parameters measured by various methods of primary clinical relevance.