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Hematologic disorders include hemoglobinopathies such as sickle-cell disease and thalassemia, anemia, and coagulopathies including hemophilia. Some of the earliest targets for gene therapy have been inherited clotting disorders, including Hemophilia A and Hemophilia B. The challenge in treating hematologic disorders with gene therapy has been achieving high enough levels for a therapeutic effect, along with maintaining stable gene expression. NAV™ Therapeutics are capable of such high-level gene delivery, which is extremely long-lasting. In this approach, the target organ is the liver, which can act as a “factory” for synthesizing and secreting clotting factors that circulate throughout the body.
Several clinical trials using gene therapy for hemophilia B have been conducted or are currently underway, including one using NAV Technology. Therapeutics for hemophilia A are at the preclinical stage.
For some of the hematologic disorders, tight control of the levels of therapeutic transgene expression will be needed for optimal efficacy and safety. In these applications, our NAV platform includes a gene regulation technology that will allow for pharmacologic control of the timing and level of gene expression.
Hematologic Disorders currently under investigation:
- Hemophilia A
- Hemophilia B
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