- REGENX Programs
- Licensed Programs
- Investigator Sponsored Programs
The challenge in treating metabolic disorders with gene therapy has been achieving high enough levels for a therapeutic effect, along with stable gene expression. NAV™ Therapeutics are capable of such high-level gene delivery, which is extremely long-lasting.
REGENX’s approach initially is to treat metabolic disorders in which the liver is the target organ for gene delivery, either for metabolic correction of liver cells, or to use the liver as a “factory” for synthesizing and secreting enzymes that circulate throughout the body.
Current status of gene therapy for metabolic disorders:
Gene therapy approaches for metabolic disorders are currently in preclinical studies. Target diseases include lysosomal storage diseases, disorders of cholesterol metabolism, Crigler-Najjar syndrome, and diabetes.
Metabolic Disorders currently under investigation:
- Familial Hypercholesterolemia
- Hurler Syndrome (MPS I)
- Maroteaux-Lamy Syndrome (MPS VI)
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