Drug intelligence / Profile preview

VX-017

Development stage
Phase 3
Lead developer
Vertex
Modality
Stem Cell Therapies → Cell Therapies
Administration
Intravenous
01

Overview

VX-017 is an investigational, allogeneic, stem cell-derived, fully differentiated islet cell therapy being developed by Vertex Pharmaceuticals for the treatment of type 1 diabetes (T1D). Designed as a "type O" or universal-donor cell product, VX-017 is engineered to be compatible with patients of all blood types, potentially doubling the addressable patient population compared to blood type-restricted islet cell therapies like zimislecel (VX-880). Like zimislecel, VX-017 consists of glucose-responsive, insulin-producing islet cells that are infused into the hepatic portal vein to restore endogenous insulin production and regulate blood glucose levels, offering a potential one-time functional cure for patients with T1D, particularly those with impaired hypoglycemic awareness and severe hypoglycemia.

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