ABO-401

**ABO-401** is an investigational gene therapy being developed by Abeona Therapeutics for the treatment of cystic fibrosis (CF). It is designed to deliver a functional, regulatable human mini-CFTR gene into the lungs using a next-generation adeno-associated virus (AAV) vector, specifically the proprietary AIM AAV204 capsid. The therapy aims to restore chloride channel function in airway epithelial cells for all CF mutations, including delta-F508, by directly targeting the underlying genetic defect. Preclinical studies show ABO-401 delivers robust expression of functional CFTR in the airways of CF mice and restores CFTR activity in human CF nasal and bronchial epithelial cells. The delivery vector, AIM AAV204, enhances gene delivery and expression in lung tissue compared to previous AAV vectors[1][2][3][7][11].