Zimislecel (VX-880): A Comprehensive Review of an Investigational Allogeneic Stem Cell-Derived Islet Cell Therapy for Type 1 Diabetes Mellitus

1. Introduction to Zimislecel (VX-880)

1.1. Overview and Therapeutic Rationale in Type 1 Diabetes (T1D)

Zimislecel, formerly designated VX-880, is an investigational advanced therapy medicinal product currently under development by Vertex Pharmaceuticals.[1] It is classified as an allogeneic, stem cell-derived, fully differentiated pancreatic islet cell therapy.[1] The core therapeutic principle underlying Zimislecel is the replacement of insulin-producing pancreatic beta cells that are selectively destroyed by an autoimmune process in individuals with Type 1 Diabetes (T1D).[2] By transplanting functional islet cells, Zimislecel aims to restore the body's capacity for endogenous insulin production, potentially obviating the need for lifelong exogenous insulin administration.[2] This approach directly targets the fundamental cellular deficit in T1D, a contrast to current standard-of-care treatments, such as exogenous insulin, which primarily manage the symptoms of hyperglycemia without addressing the autoimmune destruction of beta cells.[1]