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Gene editing could cure sickle cell and thalassemia in new trial

NCT ID NCT06647979

Recruiting now ⭐️ CURE ⭐️ Sponsor: Daniel Bauer Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage trial tests a gene-editing treatment for people with severe sickle cell disease or beta-thalassemia. The approach uses the patient's own blood stem cells, which are edited to boost fetal hemoglobin—a healthy type that can replace the defective hemoglobin. Up to 10 participants aged 13-40 will receive the edited cells after mild chemotherapy, with the goal of curing their condition without the need for a donor or lifelong medication.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

beta thalassemia beta-thalassemia major sickle cell disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

BETA-THALASSEMIA SICKLE CELL ANEMIA (HBSS, OR HBSΒ-THALASSEMIA0) SICKLE CELL DISEASE TRANSFUSION DEPENDENT BETA-THALASSAEMIA

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Boston Children's Hospital

    RECRUITING

    Boston, Massachusetts, 02115, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••