# Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Sickle Cell Disease

> **NCT07708350** · PHASE1 · NOT_YET_RECRUITING · sponsor: **Daniel Bauer** · enrollment: 5 (estimated)

## Conditions studied

- Sickle Cell Disease
- Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)

## Interventions

- **BIOLOGICAL:** autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein
- **DEVICE:** Sequencing Assay for Variant rs114518452

## Key facts

- **NCT ID:** NCT07708350
- **Lead sponsor:** Daniel Bauer
- **Sponsor class:** OTHER
- **Phase:** PHASE1
- **Study type:** INTERVENTIONAL
- **Status:** NOT_YET_RECRUITING
- **Start date:** 2026-08
- **Primary completion:** 2028-12
- **Final completion:** 2030-12
- **Target enrollment:** 5 (ESTIMATED)
- **Last updated:** 2026-07-16


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT07708350

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT07708350, "Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Sickle Cell Disease". Retrieved via MORVS 2026-10-20 from https://api.morvs.ai/clinical/NCT07708350. Licensed CC0.

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