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Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease

Studying BAH243 gene therapy for sickle cell disease treatment.

Recruiting
2-90 years
All
Phase 1/2

This study is looking at a new way to treat sickle cell disease (SCD), a condition where red blood cells are shaped like a sickle and can cause pain and other health issues. The treatment involves using a special gene therapy called **BAH243**. This study is open to people aged 2 to 50 years old with SCD.

Participants will be part of the study for up to 2 years after their stem cell transplant. They might also be asked to join a longer follow-up study for 13 more years. This will help doctors understand how safe and effective the treatment is over time. The study is designed to be as safe as possible, with careful checks before moving on to new participants.

  • The study lasts up to 15 years in total.
  • Participants must have specific types of SCD and meet certain health conditions.
  • There are potential risks and benefits, which should be discussed with your doctor.
Study details
    Sickle Cell Disease
    Sickle-Cell Disease With Crisis

NCT06399107

Essen Biotech

17 April 2026

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A clinical trial is a study designed to test specific interventions or treatments' effectiveness and safety, paving the way for new, innovative healthcare solutions.

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