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VX-264

Cell therapy (Stem-cell derived) · Cell replacement · developed by VERTEX PHARMACEUTICALS INC / MA

Phase 1/2 Most advanced trial

VRTX Developer

1 Registered trials

What is VX-264?

VX-264 is an allogeneic human stem cell-derived islet cell therapy being studied in Type 1 Diabetes. It aims to replace the insulin-producing beta cells that are lost or dysfunctional in the disease.

Summary written with AI from the sources listed at the end of this page.

At a glance

Drug type: Cell therapy (Stem-cell derived)
Mechanism: Cell replacement
Therapy area: Endocrine
Developer: VERTEX PHARMACEUTICALS INC / MA (VRTX)
FDA status: Investigational, not approved in the US. Most advanced trial: Phase 1/2

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT05791201	Phase 1/2	Type 1 Diabetes	Active not recruiting	VRTX

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

Type 1 Diabetes

More from VERTEX PHARMACEUTICALS INC / MA

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Sources

ClinicalTrials.gov NCT05791201

Updated Oct 3, 2026. Something wrong or missing? Report it on the VRTX page.

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Data: FDA (Drugs@FDA, Purple Book, DailyMed), NIH (NCI Thesaurus, GSRS, RxNorm), ClinicalTrials.gov • Informational purposes only, not medical or investment advice. • All drugs A–Z