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CRISPR stem cell therapy shows promise for kids with sickle cell

NCT ID NCT05329649

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

First seen Jun 27, 2026 · Last updated Jul 02, 2026 · Updated 2 times

Summary

This phase 3 trial tests a single dose of CTX001, a CRISPR-edited stem cell therapy, in 13 children with severe sickle cell disease who have not responded to standard treatments. The goal is to see if it can prevent severe pain crises for at least a year. Participants receive their own modified stem cells after chemotherapy to prepare the body.

What this could mean

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

Active substance
CTX001 (CRISPR-edited stem cells)
What this could lead to
If it works, this could offer children with severe sickle cell disease a long-term reduction in painful crises and hospital stays.
What could go wrong
This is a small, early-phase study (13 kids) and requires intensive chemotherapy before the infusion, which carries serious risks. Long-term effects are unknown.

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Conditions

The condition(s) this trial relates to.

hematologic disorder hemoglobinopathy inherited hemoglobinopathy sickle cell disease

As listed by the trial registrant

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

HEMATOLOGICAL DISEASES HEMOGLOBINOPATHIES HYDROXYUREA FAILURE HYDROXYUREA INTOLERANCE SICKLE CELL DISEASE

Contacts and locations

Locations

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

    Rome, Italy

  • Levine Children's Hospital - Hematology

    Charlotte, North Carolina, 28203, United States

  • St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105, United States

  • St.Mary's Hospital - Haematology Dept

    London, United Kingdom

  • The Children's Hospital of Philadelphia - Hematology

    Philadelphia, Pennsylvania, 19104, United States

  • TriStar Medical Group Children's Specialists - Pediatric Oncology

    Nashville, Tennessee, 37203, United States

  • University Hospital Duesseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology

    Düsseldorf, Germany

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