How it works
This therapy works as follows:
- Doctors collect hematopoietic stem cells from the patient’s bone marrow.
- In a specialized cleanroom, the lentiviral vector is added to the patient’s stem cells. The vector delivers the healthy gene to these cells.
- The patient receives a preparative treatment to make space in the bone marrow for the modified stem cells.
- The patient is given their own modified stem cells back, administered into the bloodstream.
- The stem cells travel to the bone marrow, where they remain and produce various types of blood cells.
- The newly formed blood cells contain the healthy gene and can travel to different parts of the body.
How is lentiviral gene therapy used in lysosomal storage disorders?
Once the corrected cells are returned to the patient, they engraft in the bone marrow, where they begin to produce and secrete the missing enzyme. These cells also differentiate into the various types of blood cells, which can travel through the bloodstream to different parts of the body, where they secrete the missing enzyme. This enzyme can then be taken up by neighboring cells and break down the molecules that have accumulated in those cells—sugars in the case of Pompe and Hunter—thereby preventing these molecules from causing damage to organs and tissues.
A unique feature of our technology is that the modified cells also reach the brain and can halt disease progression there; this is generally not the case with other forms of gene therapy.
This therapy offers the promise of a long-term, potentially lifelong solution. However, this must first be demonstrated in clinical studies. This contrasts with enzyme replacement therapies, which require lifelong infusions. This promising new approach has already been successfully applied in other diseases, such as metachromatic leukodystrophy (MLD). LentiCure aims to apply this approach to Pompe disease, Hunter syndrome, and other lysosomal storage disorders.
