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Gene editing offers hope for a One-Time sickle cell cure

NCT ID NCT03745287

Cure Sponsor: Vertex Pharmaceuticals Incorporated Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Sep 16, 2026 · Last updated Sep 17, 2026 · Updated 1 time

Summary

Researchers are testing a one-time gene therapy called exa-cel for people with severe sickle cell disease. The treatment uses CRISPR-Cas9 to edit a patient's own blood stem cells, which are then infused back after chemotherapy. The trial measures whether participants remain free of severe pain crises for at least a year and monitors safety.

What this could mean

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

Active substance
Exagamglogene autotemcel (exa-cel), a CRISPR-Cas9 gene-edited stem cell therapy
What this could lead to
If it works, this one-time treatment could free people with severe sickle cell disease from lifelong pain crises and hospital stays.
What could go wrong
The trial is small and early, and the chemotherapy conditioning carries serious risks like infertility and infection. The gene editing may not work for everyone, and long-term effects are unknown.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

63 people

The number who actually took part.

Started

Nov 2018

Finished

Jul 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 to 35 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * Diagnosis of severe sickle cell disease as defined by: * Documented severe sickle cell disease genotype * History of at least two severe vaso-occlusive crisis events per year for the previous two years prior to enrollment * Eligible for autologous stem cell transplant as per investigators judgment Key Exclusion Criteria: * An available 10/10 human leukocyte antigen (HLA)-matched related donor * Prior hematopoietic stem cell transplant (HSCT) * Clinically significant and active bacterial, viral, fungal, or parasitic infection Other protocol defined inclusion/exclusion criteria may apply.

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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 Sickle cell disease

Contacts and locations

Locations

  • Ann & Robert Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Columbia University Medical Center (21+ years)

    New York, New York, 10032, United States

  • Columbia University Medical Center (≤21 years)

    New York, New York, 10032, United States

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

    Rome, Italy

  • Hopital Necker Enfants Malades

    Paris, France

  • Hopital Universitaire des Enfants Reine Fabiola (HUDERF)

    Brussels, Belgium

  • Imperial College Healthcare NHS Trust, Hammersmith Hospital

    London, United Kingdom

  • Lucile Packard Children's Hospital of Stanford University

    Palo Alto, California, 94304, United States

  • Methodist Children's Hospital/Texas Transplant Institute

    San Antonio, Texas, 78229, United States

  • Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine, Paediatric Haemotology, Oncology and Stem Cell Transplantation

    Regensburg, Germany

  • Royal London and St Bartholomew's Hospital, Pathology and Pharmacy Building

    London, United Kingdom

  • St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105, United States

  • 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 Hospital Duesseldorf

    Düsseldorf, Germany

  • University of Illinois at Chicago Hospitals and Health Systems

    Chicago, Illinois, 60612, United States

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