Exploration of Endocrine and Metabolic Diseases

Table 1. Early clinical outcomes of stem-cell-derived β-cell replacement therapies.

Study/Program	n	Follow-up	Baseline vs. post-treatment C-peptide	Insulin reduction	HbA1c change
ViaCyte PEC-Encap (VC-01) [18]	~20 (early-phase cohorts)	Up to 12–24 months	Baseline: undetectable → Post-treatment: generally low or undetectable due to limited engraftment	No sustained insulin independence; minimal or no durable reduction	No consistent HbA1c improvement reported
ViaCyte PEC-Direct (VC-02) [18]	~17	Up to 12 months	Baseline: undetectable → Post-treatment: detectable C-peptide in a subset of patients	Partial insulin dose reductions in selected individuals	Modest HbA1c reductions in responders
Vertex VX-880 [19, 87]	n = 12 (actual phase 1/2 cohort); specific data	90–360 days	Baseline: undetectable → Post-treatment: robust fasting and stimulated C-peptide	Insulin independence in 2/6 at 1 year	HbA1c achieved levels < 7.0% to near-normal range (70–180 mg/dL)
Vertex VX-264 (encapsulated) [19, 87]	Phase 1 ongoing	Interim	Data pending	Data pending	Data pending

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Reported outcomes reflect early-phase, small-cohort studies with limited follow-up; results should be interpreted cautiously, as long-term durability, safety, and generalizability remain under investigation. HbA1c: glycated hemoglobin.

Declarations

Author contributions

MKS, CF, and IB: Conceptualization, Investigation, Writing—original draft, Writing—review & editing. KM: Conceptualization, Investigation, Conceptualization. IG, SPL, KD, KA, NK, AT, CC, and KT: Formal analysis, Investigation, Writing—review & editing. All authors read and approved the submitted version.

Conflicts of interest

The authors declare that there are no conflicts of interest.

Ethical approval

Not applicable.

Consent to participate

Not applicable.

Consent to publication

Not applicable.

Availability of data and materials

Not applicable.

Funding

Not applicable.

Copyright

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Open Exploration maintains a neutral stance on jurisdictional claims in published institutional affiliations and maps. All opinions expressed in this article are the personal views of the author(s) and do not represent the stance of the editorial team or the publisher.

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