The Next Frontier: Eliminating Immunosuppressants
Because VX-880 requires lifelong immunosuppressive drugs, its direct reach will focus on approximately 60,000 brittle T1D patients who face immediate risks from hypoglycemia unawareness.
To bring stem-cell islets to the broader T1D population, Vertex is advancing parallel pipelines:
1. VX-264: Uses the identical stem-cell islet cells as VX-880, but houses them inside a surgically implanted, retrievable encapsulation device. The semi-permeable membrane allows oxygen, glucose, and insulin to diffuse freely while physically blocking immune cells and antibodies, eliminating the need for immunosuppressive medication.
2. Hypoimmune (Gene-Edited) Islets: In partnership with Sana Biotechnology, developing “stealth” stem-cell islets where specific HLA genes are knocked out and CD47 is overexpressed, disguising the cells from both innate and adaptive immune recognition without mechanical capsules or systemic drugs.
Detailed enrollment criteria and verified site listings are maintained on ClinicalTrials.gov (NCT04786262).