AAV5-FTH1

AAV5-FTH1 is an experimental gene therapy that utilizes an adeno-associated virus serotype 5 (AAV5) vector to deliver the Ferritin Heavy Chain 1 (FTH1) gene. FTH1 is a critical component of ferritin, the primary intracellular protein responsible for sequestering iron and maintaining iron homeostasis. The therapy is designed to overexpress FTH1 in target tissues, such as alveolar epithelial cells, to mitigate iron ion disorders and prevent ferroptosis—a form of regulated cell death driven by iron-dependent lipid peroxidation. In preclinical models of chronic obstructive pulmonary disease (COPD) and smoking-induced emphysema, AAV5-FTH1 has demonstrated the ability to restore iron balance and protect lung tissue from oxidative damage and emphysematous changes.

Other names

AAV5-Ferritin Heavy Chain 1AAV-5-Ferritin Heavy Chain 1AAV 5-Ferritin Heavy Chain 1Adeno-associated virus serotype 5-FTH1