June 2020
Volume 61, Issue 7
Free
ARVO Annual Meeting Abstract  |   June 2020
Phase I/IIa Clinical Trial of Human Embryonic Stem Cell (hESC)-Derived Retinal Pigmented Epithelium (RPE, OpRegen) Transplantation in Advanced Dry Form Age-Related Macular Degeneration (AMD): Interim Results
Author Affiliations & Notes
  • Christopher D Riemann
    Cincinnati Eye Institute, Cincinnati, Ohio, United States
  • Eyal Banin
    Department of Ophthalmology, Center for Retinal and Macular Degenerations, Jerusalem, Israel
  • Adiel Barak
    Department of Ophthalmology, Sourasky Medical Center, Tel Aviv, Israel
  • David S Boyer
    Retina Vitreous Associates Medical Group, Los Angeles, California, United States
  • Rita Ehrlich
    Department of Ophthalmology, Rabin Medical Center, Petah Tikva, Israel
  • Tareq Jaouni
    Department of Ophthalmology, Center for Retinal and Macular Degenerations, Jerusalem, Israel
  • Richard McDonald
    West Coast Retina Group, San Francisco, California, United States
  • David Telander
    Retinal Consultants Medical Group, Sacramento, California, United States
  • Michael Keane
    Gyroscope Therapeutics, Ambler, Pennsylvania, United States
  • Jessica Ackert
    Gyroscope Therapeutics, Ambler, Pennsylvania, United States
  • Mark D. Ferguson
    Gyroscope Therapeutics, Ambler, Pennsylvania, United States
  • Avi Ben- Shabat
    Lineage Cell Therapeutics, Inc., (subsidiary, Cell Cure Neurosciences), Jerusalem, Israel
  • Jordi Mones
    Institut de la Màcula, Barcelona, Spain
  • Diana Angelini
    Lineage Cell Therapeutics, Inc., Carlsbad, California, United States
  • Gary S. Hogge
    Lineage Cell Therapeutics, Inc., Carlsbad, California, United States
  • Benjamin Reubinoff
    Center for Embryonic Stem Cells and the Department of Gynecology and Obstetrics, Hadassah-Hebrew University Medical Center, Jerusalem, Israel
  • Footnotes
    Commercial Relationships   Christopher Riemann, Alcon (C), Alimera (C), Allergan (C), Aniridia Foundation International (C), Bausch & Lomb/Valeant (C), BMC/Eyetube (C), Chruman Research (I), Clovernook Center for the Blind and Visually Impaired (S), CSTLII (C), CVP (CEI Vision Partners) (I), Digital Surgery Systems (I), Gore (C), Gyroscope Therapeutics (Orbit Biomedical) (C), Gyroscope Therapeutics (Orbit Biomedical) (P), Haag Streit AG (C), Haag Streit Surgical (C), Haag Streit USA (P), HumanOptics AG (C), Iamc2 (P), iVeena (I), Janssen / Johnson & Johnson (P), Kaleidoscope Engineering (P), Macor Industries (I), MedOne (I), Northmark Pharmacy (I); Eyal Banin, 20180008458; 20150125506; 20150118749; 20110177594; 20110027333; 20090104695 (P), Lineage Cell Therapeutics, inc. (subsidiary, Cell Cure Neurosciences, Ltd.) (C); Adiel Barak, None; David Boyer, Lineage Cell Therapeutics, Inc. (C); Rita Ehrlich, None; Tareq Jaouni, None; Richard McDonald, None; David Telander, None; Michael Keane, Gyroscope Therapeutics (E); Jessica Ackert, Gyroscope Therapeutics (E); Mark Ferguson, Gyroscope Therapeutics (E); Avi Shabat, Lineage Cell Therapeutics, Inc., (subsidiary, Cell Cure Neurosciences) (E); Jordi Mones, Lineage Cell Therapeutics, Inc., (subsidiary, Cell Cure Neurosciences) (C); Diana Angelini, Lineage Cell Therapeutics Inc. (E); Gary Hogge, Lineage Cell Therapeutics, Inc. (E); Benjamin Reubinoff, 10,377,989; 10,370,644; 10,174,285; 10,066,203; 9,969,973; 9,951,314; 9,862,925; 9,834,751; 9,752,126; 9,658,216; 9,650,605; 9,506,036; 9,439,932; 9,243,225; 9,234,178; 9,005,965; 8,956,866; 8,940,537; 8,597,947; 8,367,406; 8,257,973; 7,780,993; 7,682,826; 6,921,632; 6,875,607 (P), Lineage Cell Therapeutics, Inc., subsidiary, (Cell Cure Neurosciences) (C)
  • Footnotes
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Investigative Ophthalmology & Visual Science June 2020, Vol.61, 865. doi:
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      Christopher D Riemann, Eyal Banin, Adiel Barak, David S Boyer, Rita Ehrlich, Tareq Jaouni, Richard McDonald, David Telander, Michael Keane, Jessica Ackert, Mark D. Ferguson, Avi Ben- Shabat, Jordi Mones, Diana Angelini, Gary S. Hogge, Benjamin Reubinoff; Phase I/IIa Clinical Trial of Human Embryonic Stem Cell (hESC)-Derived Retinal Pigmented Epithelium (RPE, OpRegen) Transplantation in Advanced Dry Form Age-Related Macular Degeneration (AMD): Interim Results. Invest. Ophthalmol. Vis. Sci. 2020;61(7):865.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose : Introduction of healthy RPE cells may be of therapeutic benefit in dry AMD patients. We developed technology to derive RPEs from hESCs using cGMP directed differentiation. Safety and tolerability of these cells is being evaluated in a Phase I/IIa clinical study in patients with dry AMD and geographic atrophy (GA) (NCT02286089). We report accumulated safety and imaging data from all subjects in the fully-enrolled first 3 cohorts (n=12) and ongoing 4th cohort.

Methods : Subretinal transplantation of 50-200k OpRegen cells in suspension to the worse vision eye uses either pars plana vitrectomy (PPV) and retinotomy or the Orbit subretinal delivery system (SDS) under local anesthesia. Short course, perioperative systemic immunosuppression is used. Systemic and ocular safety is closely observed, and retinal function and structure are monitored using various imaging modalities.

Results : Cohorts 1-3 are in long-term follow-up. Cohort 4 dosing is ongoing. Treatment has been well tolerated to date, with no unexpected adverse events (AEs) or treatment-related systemic serious adverse events reported. Using PPV, the most common ocular AEs were the formation of predominately mild epiretinal membranes (ERM), though two severe ERM were surgically peeled. Additionally, one PPV-treated patient experienced a retinal detachment. All 3 events were successfully treated. AEs associated with the Orbit SDS include eyelid edema and subconjunctival hemorrhage (n=1, each). Visual improvement has been noted in all cohort 4 patients to date (n=4; 10-22 letters), which has been maintained from 4.5 to over 15 months. In several subjects, within the area of RPE cell transplant, improvements of the ellipsoid zone and RPE layers at the border of GA, as well as directional growth changes in the area of GA, have been seen. Persistent changes observed following treatment include, alterations in drusen appearance, subretinal pigmentation and hyper-reflective areas, suggestive of the presence of transplanted RPE cells.

Conclusions : Subretinal transplantation of hESC-derived RPE cells in patients with dry AMD and GA appears well tolerated. Imaging findings suggest presence of transplanted cells in the subretinal space. Encouraging structural and clinical changes observed in some patients will require additional follow-up.

This is a 2020 ARVO Annual Meeting abstract.

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