A New Take on Established Science
Having been under development for almost 20 years at two of the leading academic medical and research institutions in Europe, APR-2020’s cellular gene therapy platform utilizes two established and trusted methods combined in a unique way to create a novel therapy.
It is based on proven scientific and clinical principles that have been used successfully in a number of approved cellular gene therapies.
In the case of APR-2020, autologous CD34+ cells are transduced with a lentiviral vector that has been embedded with a corrected target gene.
Lentiviral vectors work by inserting, modifying, or removing genes in host cells.
With APR-2020, the lentiviral vector integrates a corrected copy of the RPS19 gene into the defective red blood cell-generating stem and progenitor cells.
The inserted genes increase the expression of the final protein product, which reduces the amount of RPS19 gene transduction necessary for the corrected patient cells to engraft into the patient’s bone marrow, begin erythropoiesis and normalize cellular activity.
Once the defective red blood cell-generating stem and progenitor cells have been transduced with a working copy of the RPS19 gene, they are infused back into the patient. There, they can begin regular bone marrow activity and red blood cell production.