Benzinga | Apr 20, 2021 07:40AM EDT

Abeona Therapeutics To Highlight New Data From Preclinical Research At Association For Research In Vision And Ophthalmology 2021 Annual Meeting May 1-7

Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that new data from its preclinical research will be highlighted during an oral presentation at the Association for Research in Vision and Ophthalmology (ARVO) 2021 Annual Meeting, being held virtually from May 1-7, 2021.



"Autosomal recessive Stargardt disease is caused by mutations in the ABCA4 gene, preventing removal of toxic substances from photoreceptor cells that result in photoreceptor death and progressive vision loss," said Linas Padegimas, Ph.D., Senior Director, Product Development at Abeona. "The preclinical data being presented at ARVO's Annual Meeting provides compelling evidence that two independent AAV vectors utilizing Cre recombinase can efficiently reconstitute the ABCA4 gene, leading to full-size ABCA4 protein expression. Future studies that explore Cre-mediated ABCA4 gene reconstitution and lipofuscin clearance in vivo could provide additional findings to inform clinical development plans for a dual AAV vector approach as a potential treatment for Stargardt disease. In addition, our Cre-mediated dual AAV vector technology holds potential benefit for other indications that require delivery of larger genes that exceed the encapsidation capacity of AAV vectors."

Details of the oral presentation are as follows:

Title: Dual AAV Vector Strategy for Expression of Large Genes Targeted for Stargardt Disease Gene Therapy Development

Presenter: Dr. Brianna Barrett, Abeona Therapeutics Inc.

Session Title: Gene therapy in ocular diseases

Session Date and Time: May 4, 2021 from 2:15 PM to 3:45 PM EDT