Abeona Reports Preclinical Data of Experimental CF Gene Therapy

April 30, 2019

Abeona Therapeutics said new preclinical data on its experimental gene therapy for cystic fibrosis, ABO-401, show it efficiently delivered a highly-expressed, functional copy of human mini-CFTR gene to the lung in mouse models and restored function in human cystic fibrosis patient nasal and bronchial epithelial cells with the most common mutation of the gene.

The data will be presented at the American Society of Gene and Cell Therapy 22nd Annual Meeting in Washington, D.C.

ABO-401 is a novel gene therapy in development for the treatment of patients with cystic fibrosis (CF), a progressive genetic disease that results in persistent lung infections and limits the ability to breathe over time. It is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CTFR) gene. ABO-401 targets airway cells and corrects the underlying CF chloride channel deficit and addresses all CF mutations, including the most common CF mutation, delta-F508.

ABO-401 has a human mini-CFTR gene that can be regulated and that is 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. AAV204 is one of the company’s next generation of edeno-associated virus capsids for use in gene therapies.

The new data to be presented demonstrated that ABO-401 efficiently delivered a highly-expressed, functional copy of CFTR 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 a 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.

“These encouraging preclinical data add to the growing body of evidence suggesting ABO-401 may address the challenges in lung delivery and transgene expression that have limited the advancement of gene therapy for CF patients,” said Timothy Miller, president and chief scientific officer of Abeona.

Miller said ABO-401 is being advanced into studies that will enable the filing of an application to begin human clinical trials. If successful, the treatment could provide a one-time gene therapy to address CF.

Photo: Timothy Miller, president and chief scientific officer of Abeona

Author: Rare Daily Staff

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