Cell Cure Neurosciences Ltd. (Cell Cure) is focused on the development of cell therapies for retinal and neural degenerative diseases. Its therapeutic cells include retinal pigmented epithelial (RPE) cells and neural progenitor cells. Both of these cells are derived from human embryonic stem cells (hESCs), which are considered to be the gold standard source of cells for the development of stem cell therapies. The company's OpRegen™ product is intended for use in RPE transplantations in patients with age related macular degeneration (AMD) and is expected to be among the first hESC based products to reach clinical trials.
Sector and Field of Activity
The dry form of AMD is the leading cause of visual impairment over the age of 55. There is no cure at present. The most promising future therapy is the replacement of the layer of damaged RPE cells that support and nourish the retina. In RPE transplantation cells have been obtained from other regions of the diseased eye, fetal and adult donor tissue and various cell lines. However, the lack of a reliable and ample supply of healthy RPE cells has hindered the development of RPE transplantation as a therapeutic approach to AMD. RPE cells derived from human embryonic stem cells are considered to be best, since in principle they can be produced in the homogeneous, large quantities required from a cell therapy product. Until now researchers have had to rely on the spontaneous differentiation of hESCs into RPE cells that occurs in only a few hESC lines.To achieve the full potential of hESC stem cells for the production of RPE cells, a reliable, driven differentiation method is required A second challenge facing the field involves the way RPE cells are transplanted. In animal studies clumps or suspensions of cells are injected below the macular. For use in humans, RPE cells need to be mounted on a membrane to prevent transplanted cells from migrating, clumping, forming multilayers and causing retinal detachment. This membrane replaces the Bruch membrane that is destroyed by disease or prior surgery.
Science and Technology
Cell Cure's lead OpRegen™ cell therapy product utilizes two new technologies that address these concerns. First, Cell Cure uses a new method (WO2008129554) developed by scientists at Hadassah University Hospital that drives the differentiation of human embryonic stem cells into RPE cells. These researchers have shown in a small animal model of AMD that RPE cells produced using this method can preserve vision when transplanted below the retina. Second, Cell Cure is utilizing a novel technology to produce ultrathin membranes of a biologically compatible material coated with a polarized monolayer of its hESC derived RPE cells. Over the next year, the company plans to test the cell membrane complex in a large animal model. Cell Cure's other cell therapy products under development include neural progenitor cells designed to replace the dopamine producing cells destroyed in Parkinson's disease (PD) and Cell Cure's NeurArrest™ neural cells that target in on and modulate the immune system's self-destruction of myelin coating of nerve cells in multiple sclerosis..
Key Milestones for 2009-2010
- Selection of the clinical grade hESCs as starting material
- Completion of the development of cryopreservation methods for RPE cells
- Successful in vitro testing of RPE cells bound to a biocompatible membrane
- Demonstration of safety and efficacy in a porcine animal model
- Start of production of clinical grade cells for clinical trials
As soon as proof of concept is obtained in the large animal study, the production of clinical grade cells for use in clinical trials will begin. The company will continue on a limited basis its development work on neural progenitor cells intended for use in Parkinson's disease and multiple sclerosis. The achievement of these goals depends on new funding.
Team – Management and Scientific Advisors
By combining Cell Cure's product development capabilities with cutting edge technology from Hadassah University Hospital's Human Embryonic Stem Cell Research Center and the Departments of Ophthalmology and Neurology, the company is well positioned to be among the first to reach clinical trials. The company's R&D activities are currently carried out by a team of 15 scientists with laboratories located on the campus of Hadassah University Hospital in close proximity to the hospital’s Human Embryonic Stem Cell Research Center. Prof. Benjamin Reubinoff serves as Cell Cure’s CSO and directs its R&D activities. He is one of the pioneers of hESC research and is a leading researcher in the area of hESC derived neural progenitor cells. Prof. Reubinoff has successfully partnered with Prof. Tamir Ben-Hur, an expert in the field of stem cell therapy for neurodegenerative diseases and Prof. Eyal Banin, an expert in the field of cell therapy for retinal and macular degenerations, to produce landmark papers. Both serve as consultants to Cell Cure. The excellence of Cell Cure's scientific team has been recognized by grants from the Michael J. Fox Foundation, the IsrA.L.S Foundation, the European Community, and the Israeli government Industrial R&D programs. Charles S. Irving, Ph.D. serves as CEO and brings to the company extensive experience in the biomedical and biotech fields. Prior to joining Cell Cure he successfully led a biotech company through development and clinical studies to the out-licensing of its product to a major pharmaceutical company.
Shareholders
Cell Cure's shareholders include ES Cell International Pte Ltd (55%), Hadasit Bio-Holdings Ltd. (34%) and Teva Pharmaceutical Industries (11%). Both ES Cell International and Hadasit are potential suppliers of key clinical grade hESCs needed for producing clinical grade RPE cells.
Intellectual Property Status
ES Cell International has exclusively licensed to Cell Cure all of its IP related to neurodegenerative diseases, which includes three important patent families covering methods and cells related to the derivation of neural cells from hESCs, as well as their transplantation. The company is also developing its own IP, as well as in-licensing IP from Hadasit Ltd.
Investment Sought
Cell Cure is seeking an investment of $10M to be used to advance its OpRegen™, product through initial clinical studies and to continue the development of its product for Parkinson's disease.
