In short
DREAM01 is an investigational gene therapy being studied for sickle cell disease, a condition affecting red blood cells. It is given as a single intravenous infusion of a patient's own modified blood stem cells. One authorised Phase 1/2 trial is currently testing its efficacy and safety in 13 participants with severe sickle cell disease.
Key points
- DREAM01 is an investigational gene therapy made from a patient's own modified blood stem cells.
- It is being studied in sickle cell disease, a condition where red blood cells become sickle-shaped.
- The single authorised trial is in Phase 1/2 and plans to enrol 13 participants.
- The treatment is given as a single intravenous infusion of genetically modified stem cells.
- The trial measures efficacy and safety after the infusion, with or without a prior course of imatinib.
- The trial is currently authorised and is being run by Assistance Publique - Hôpitaux de Paris.
What is DREAM01?
DREAM01 is an investigational gene therapy, which means it has no marketing authorisation and is still being studied in clinical trials. It is a cell therapy made from a person's own blood stem cells, known as autologous CD34+ cells, which are collected and then genetically modified in a laboratory before being given back to the patient. The product record lists the pharmaceutical form as a dispersion for infusion and the route of administration as intravenous infusion. The trial is being run by Assistance Publique - Hôpitaux de Paris.
How does DREAM01 work?
In sickle cell disease, a faulty version of the haemoglobin gene leads to the production of an abnormal form of haemoglobin called beta-S globin. DREAM01 is designed to correct this at the genetic level. The patient's own blood stem cells are modified outside the body using a lentiviral vector, a tool that delivers genetic material into cells. This vector carries a corrected beta-globin gene (beta-AS3) and a small piece of genetic material called a micro-RNA (miR7m) that specifically targets and reduces the faulty beta-S globin messenger RNA. The aim is to restore the production of normal haemoglobin in red blood cells.
Which conditions is DREAM01 being studied for?
- Sickle cell disease (SCD) — a blood condition in which red blood cells become rigid and sickle-shaped, causing pain, anaemia and organ damage. The trial focuses on patients with severe forms of the disease.
How is DREAM01 given?
DREAM01 is prepared as a dispersion for infusion and is given as a single intravenous infusion. The cells are collected from the patient, modified in the laboratory, and then returned through a vein. In some participants, the trial also includes a prior course of imatinib, an anti-inflammatory medicine taken by mouth, when severe inflammation is detected at the start of the study.
What do the clinical trials show?
There is one authorised clinical trial of DREAM01, registered in the EU Clinical Trials Information System under the number 2025-521257-17-00. It is a Phase 1/2 open-label cohort comparative study, meaning that participants and researchers know which treatment is being given, and different groups are compared. The trial plans to enrol 13 participants with severe sickle cell disease.
The main thing the trial measures is efficacy and safety after the intravenous infusion of DREAM01, with or without prior administration of imatinib. Efficacy means how well the treatment works, and safety means what side effects it causes. The trial also looks at whether the genetic modification restores normal red blood cell production.
The trial is currently authorised, which means it has received approval to start recruiting participants. The study type is interventional, meaning participants receive the treatment as part of the research.
What side effects are known?
No information about side effects of DREAM01 is available from the sources provided. The trial is designed to measure safety, but results have not yet been reported in these records.