Gene therapy offers hope for sickle cell patients by boosting healthy hemoglobin
NCT ID NCT07640815
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study offers expanded access to a gene therapy for people with severe sickle cell disease. The treatment uses a modified virus to deliver genetic material that increases fetal hemoglobin, a healthy type that prevents sickling. Patients receive their own blood stem cells back after a mild chemotherapy, reducing risks like graft-versus-host disease. Early data from 36 patients show no unexpected safety issues and increased fetal hemoglobin.
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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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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Locations
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States