Efficacy Safety Study of Gene Therapy for Sickle Cell DiseaseSCD Using Autologous CD34+ Cells Transduced ex Vivo, Carrying a Corrected Globin Gene and a Silencing RNA.

Sponsor
Assistance Publique - Hôpitaux de Paris
Study ID
NCT07432867
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
12 Years - 35 Years
Healthy Volunteers
Not accepted

Interventions

  • DREAM01 drug product — GENETIC
    Each patient will receive a single IV infusion of DREAM01, autologous CD34+ stem cells transduced with βAS3m/miR7m lentiviral vector
  • anti-inflammatory therapy — DRUG
    Patient will receive anti-inflammatory therapy if necessary

Study Details

The purpose of this study is to evaluate the Safety and Efficacy of DREAM01, a gene therapy for Sickle Cell Disease (SCD). The therapy consists of transplanting autologous CD34+ cells transduced ex vivo with a bifunctional lentiviral vector expressing βAS3m-globin and an anti-βS miRNA. It aims to reduce or eliminate vaso-occlusive events and long-term organ damage in severe SCD patients lacking a Human Leukocyte Antigen (HLA) identical sibling donor.

Key Dates

First listed
Feb 25, 2026
Start date
Feb 25, 2026
Status verified
Feb 2026
Primary completion
Feb 29, 2032
Completion
Feb 28, 2033

Study Design

Enrollment
15 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: DREPAMIR drug product
    DREAM01 is a genetically modified cell therapy product that consists of autologous CD34+ cells transduced ex vivo by the bifunctional βAS3m/mR7m lentiviral vector expressing the βAS3m-globin and a micro-RNA (miRNA) targeting specifically the endogenous sickle βS-globin gene. With or without prior administration of an anti-inflammatory therapy in case of severe inflammation detected at the inclusion phase

Primary Outcome Measure

Neutrophil recovery [ Time Frame: within the 24 months following IV infusion of DREAM01 ]

Central Contacts

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