Abeona Therapeutics to report new preclinical ABO-401 data

Abeona Therapeutics will report new data demonstrating that ABO-401, the company’s gene therapy for cystic fibrosis, or CF, delivered a highly-expressed, functional copy of human mini-CFTR, or hCFTR, to the lung of CF mice and restored CFTR function in human CF patient nasal and bronchial epithelial cells. The data will be presented this evening at the American Society of Gene and Cell Therapy. ABO-401 has a regulatable human mini-CFTR gene that is efficiently packaged into one of the company’s next-generation AIM library capsids, AAV204. In this and other preclinical studies, ABO-401 restored CFTR expression and chloride conductance in airway epithelia, the main cells of the lung that contribute to CF pathology in humans. These new data demonstrated that ABO-401 delivered a highly-expressed, functional copy of hCFTR to the lungs of CF mice and restored CFTR function in nasal and bronchial epithelial cells of human donor cells with the delta-F508 mutation, the most common mutation of CF. ABO-401 transduced human CF nasal and bronchial epithelial cells, with CFTR-specific change in short-circuit current that was comparable or superior to existing modulator therapy in these same cells. Robust expression of AAV204 in the lungs of CF mice was observed and demonstrated that the AAV204 capsid was equally or more efficient at delivering gene expression cassettes to the lung compared to other naturally-occurring AAV capsids. Further, the data demonstrated that ABO-401 restored CFTR-specific nasal potential difference in CF mice, and that the ABO-401 gene expression cassette makes a fully-processed CFTR.

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