Gene therapy aims to stop sickle cell pain crises

NCT ID NCT07432867

Recruiting now Disease control Sponsor: Assistance Publique - Hôpitaux de Paris Source: ClinicalTrials.gov ↗

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

Summary

This trial tests a new gene therapy called DREAM01 for people with severe sickle cell disease who cannot find a matching bone marrow donor. The therapy uses the patient's own blood stem cells, modified to produce healthy hemoglobin and block the sickle cell gene. Up to 15 participants aged 12-35 will receive a single infusion, with the goal of reducing painful vaso-occlusive events and long-term organ damage.

What this could mean

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

Active substance: DREAM01 gene therapy (modified stem cells)
What this could lead to: If successful, this could offer a one-time treatment that reduces or eliminates painful crises and organ damage for people with severe sickle cell disease who lack a matched donor.
What could go wrong: This is an early phase 1/2 trial with only 15 participants, so results may not apply to everyone. Gene therapy carries risks like failed engraftment or side effects from the conditioning regimen.

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Conditions

The condition(s) this trial relates to.

sickle cell disease Vaso-Occlusive Crises

As listed by the trial registrant

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

SICKLE CELL DISEASE

Contacts and locations

Locations

Department of Biotherapy, Necker-Enfants Malades Hospital
RECRUITING

Paris, Île-de-France Region, 75015, France

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