CRISPR gene editing offers hope for thalassemia patients to ditch transfusions

NCT ID NCT03655678

Completed Disease control Sponsor: Vertex Pharmaceuticals Incorporated Source: ClinicalTrials.gov ↗

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

Summary

This study tested a single treatment using CRISPR gene editing to modify a person's own blood stem cells, aiming to help people with severe beta-thalassemia stop needing regular blood transfusions. 59 participants received the therapy and were monitored for safety and how long they could go without transfusions. The goal was to see if this one-time treatment could control the disease long-term.

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Conditions

The condition(s) this trial relates to.

beta thalassemia beta-thalassemia major hematologic disorder hemoglobinopathy hereditary disease inherited hemoglobinopathy thalassemia

As listed by the trial registrant

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

BETA-THALASSEMIA GENETIC DISEASES, INBORN HEMATOLOGIC DISEASES HEMOGLOBINOPATHIES THALASSEMIA

Contacts and locations

Locations

Ann & Robert Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States
British Columbia Children's Hospital

Vancouver, Canada
Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Columbia University Medical Center

New York, New York, 10032, United States
Columbia University Medical Center (21+ years)

New York, New York, 10032, United States
Imperial College Healthcare NHS Trust, Hammersmith Hospital

London, United Kingdom
Lucile Packard Children's Hospital

Palo Alto, California, 94304, United States
Ospedale Pediatrico Bambino Gesù, IRCCS

Rome, Italy
Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine

Regensburg, Germany
The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers

Nashville, Tennessee, 37203, United States
The Hospital for Sick Children

Toronto, Canada
University College London Hospitals NHS Foundation Trust

London, United Kingdom
University Hospital Tübingen

Tübingen, Germany
Universitätsklinikum Düsseldorf Hospital Duesseldorf

Düsseldorf, Germany

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