# Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

> **NCT06647979** · PHASE1 · RECRUITING · sponsor: **Daniel Bauer** · enrollment: 10 (estimated)

## Conditions studied

- Sickle Cell Disease
- Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
- Beta-Thalassemia
- Transfusion Dependent Beta-Thalassaemia

## 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:** NCT06647979
- **Lead sponsor:** Daniel Bauer
- **Sponsor class:** OTHER
- **Phase:** PHASE1
- **Study type:** INTERVENTIONAL
- **Status:** RECRUITING
- **Start date:** 2025-12-01
- **Primary completion:** 2028-12
- **Final completion:** 2030-12
- **Target enrollment:** 10 (ESTIMATED)
- **Last updated:** 2026-02-03


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT06647979

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT06647979, "Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies". Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/clinical/NCT06647979. Licensed CC0.

---

*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*