# Stem Cell Gene Therapy for Sickle Cell Disease

> **NCT02247843** · PHASE1,PHASE2 · COMPLETED · sponsor: **Donald B. Kohn, M.D.** · enrollment: 4 (actual)

## Conditions studied

- Sickle Cell Disease

## Interventions

- **BIOLOGICAL:** βAS3-FB vector transduced peripheral blood CD34+ cells

## Key facts

- **NCT ID:** NCT02247843
- **Lead sponsor:** Donald B. Kohn, M.D.
- **Sponsor class:** OTHER
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2014-12
- **Primary completion:** 2025-09-24
- **Final completion:** 2025-09-24
- **Target enrollment:** 4 (ACTUAL)
- **Last updated:** 2025-12-17

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT02247843

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT02247843, "Stem Cell Gene Therapy for Sickle Cell Disease". Retrieved via AI Analytics 2026-07-02 from https://api.ai-analytics.org/clinical/NCT02247843. Licensed CC0.

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