Business Description

Overview We are a clinical-stage biotechnology company developing cell replacement therapies to treat serious medical conditions. Certain diseases and medical events can arise from the loss of critical cellular activity and lead to devastating or difficult-to-treat conditions or impairments. Our work is grounded in the emerging evidence that replacing or supporting those cells that have become dysfunctional or “lost” (destroyed or dead) can restore or replenish normal function and improve treatment and recovery paradigms. We call this approach “Replace and Restore”. We believe cellular therapies aimed at entirely replacing dysfunctional or destroyed cells may have more durable, broader, or suitable applicability than traditional pharmaceutical products, which often seek to affect just a single molecular target or group of biological pathways. Transplantation of replacement cells represents an emerging branch of medicine, and we believe we are uniquely positioned to capitalize on its opportunities by demonstrating the value of administering mature, differentiated cells to patients. We are developing a portfolio of assets based on this mechanism and our most clinically-advanced program to date is OpRegen (RG6501), an allogeneic retinal pigmented epithelial (RPE) cell replacement therapy currently in Phase 2a development under a worldwide collaboration with F. Hoffman-La Roche Ltd. and Genentech, Inc., a member of the Roche Group (collectively or individually, “Roche” or “Genentech”), for the treatment of geographic atrophy (GA) secondary to dry-AMD. The cells we have or may seek to manufacture are intended to replace the same type of cells that the patient has lost, or which have become dysfunctional due to various conditions, such as in dry age-related macular degeneration (dry-AMD), spinal cord injury (SCI), hearing loss and Type 1 Diabetes (T1D). We view the term cell transplant as more applicable to our approach than “stem cell therapy”, because we never administer stem cells to patients. Instead, we utilize stem cells solely as a source material, from which to create and deliver partly or fully differentiated (i.e. mature) and functionally active “replacement” cells that are intended to be substantially identical to the cells which have been impacted by disease or trauma. These replacement cells are then transplanted, historically in a one-time procedure, to treat conditions caused by the loss or dysfunction of a specific cell type. Additionally, each of our product candidates is allogeneic and is derived from a single, carefully selected and cultured cell line for the life of the product, which eliminates donor variability and may mitigate certain regulatory and clinical risks. Our programs are based on our proprietary, in-house, cell-based manufacturing platform, which we call AlloSCOPE™ (Allogeneic, Scalable, Consistent, Off-the-shelf, Pluripotent Cell Engineering), and supported by our associated development, formulation, manufacturing, and delivery capabilities. The AlloSCOPE platform is a proprietary differentiation and production modality from which, i) a single, well-characterized pluripotent cell line can create a stable current Good Manufacturing Practice (cGMP) master cell bank (MCB), ii) a vial from our MCB can create a cGMP working cell bank (WCB), and iii) a vial from our WCB can create several hundred to many thousands of vials of a final, allogeneic cell-based product, ready for patient dosing. This process confers consistent, cost-effective, and scalable cell-based production. Importantly, the AlloSCOPE platform can be applied across multiple programs, which we believe could offer advantages in the pursuit of commercially successful, allogeneic and “off the shelf” cell therapies. In some instances, we also apply a proprietary “thaw-and-inject” formulation into our product profiles, which allows for rapid dosing and “immediate use” of our cells. This formulation technology can greatly reduce the lengthy dose preparation steps often associated with certain cell therapy programs.…