CRISPR stem cell therapy could end transfusions for sickle cell and thalassemia patients

NCT ID NCT05477563

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

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

Summary

This study tests a one-time treatment called CTX001, which uses CRISPR gene editing to modify a patient's own stem cells to produce more fetal hemoglobin. The goal is to reduce or eliminate the need for blood transfusions in people with transfusion-dependent beta-thalassemia or severe sickle cell disease. The trial involves 26 participants and includes a conditioning step with chemotherapy before the stem cells are infused.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: CRISPR-edited stem cells (CTX001, also called exagamglogene autotemcel)
What this could lead to: If it works, this could free people with severe sickle cell disease or beta-thalassemia from needing regular blood transfusions or lifelong medication.
What could go wrong: This is a small, early-phase 3 study with only 26 participants. The treatment requires strong chemotherapy before the infusion, which can cause serious side effects. Long-term safety and effectiveness are still unknown.

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Conditions

The condition(s) this trial relates to.

beta thalassemia beta-thalassemia major hematologic disorder hemoglobinopathy hereditary disease inherited hemoglobinopathy sickle cell disease 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 SICKLE CELL ANEMIA SICKLE CELL DISEASE THALASSEMIA

Contacts and locations

Locations

IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
RECRUITING

Rome, Italy
King Faisal Specialist Hospital & Research Centre - Riyadh - Hematology
RECRUITING

Al Mathar Ash Shamali, Saudi Arabia
Levine Children's Hospital - Hematology
RECRUITING

Charlotte, North Carolina, 28203, United States
New York Presbyterian Hospital - Morgan Stanley Children's Hospital
RECRUITING

New York, New York, 10032, United States
TriStar Medical Group Children's Specialists - Pediatric Oncology
RECRUITING

Nashville, Tennessee, 37203, United States
University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology
RECRUITING

Düsseldorf, Germany

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