Business
Abeona Therapeutics Announces Update on AAV Ophthalmology Program
Advancing AAV-based gene therapy candidates toward IND studies on encouraging animal proof of concept data in Stargardt Disease, X-linked Retinoschisis
About this update from Abeona Therapeutics Inc.
[{"type":"text","content":"Advancing AAV-based gene therapy candidates toward IND studies on encouraging animal proof of concept data in Stargardt Disease, X-linked Retinoschisis (XLRS), and Autosomal Dominant Optic Atrophy (ADOA) To submit first pre-Investigational New Drug (IND) application meeting request this month NEW YORK and CLEVELAND, March 14, 2023 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced three internally developed investigational preclinical gene therapy product candidates from its ophthalmology program. Abeona’s preclinical programs are investigating the use of novel adeno-associated virus (AAV) capsids in therapies for serious genetic eye diseases. “We are excited by the broad potential for treating serious eye diseases with new AAV-based therapies using novel AAV capsids from our in-licensed AIM™ capsid library and internal research,” said Brian Kevany, Ph.D., Chief Technical Officer and Head of Research at Abeona. “In 2022, we evaluated the ability of our gene constructs and capsids to deliver and express the recombinant protein in target eye tissues and rescue mutant phenotypes in mouse disease models. Based on encouraging findings from these animal proof of concept experiments, we are looking forward to reporting new data from these programs at a scientific congress in the second quarter of 2023 and gaining alignment with the U.S. FDA on the clinical development plans for these programs.” Preclinical Product Candidate ABO-504 for Stargardt Disease Abeona’s internal research and development team developed ABO-504, which is designed to efficiently reconstitute the full-length ABCA4 gene by implementing a dual AAV vector strategy using the Cre-LoxP recombinase system. In May 2021 at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, Abeona reported preclinical data demonstrating the ability of the dual AAV vector system to produce full length ABCA4 protein in cell culture. Recent proof-of-concept studies have extended these findings by showing expression of ABCA4 mRNA and full-length ABCA4 protein in the retina of subretinally dosed abca4-/- knockout mice, at levels similar to endogenous ABCA4 in wild-type animals. Preclinical Product Candidate ABO-503 for X-linked Retinoschisis (XLRS) ABO-503, composed of a functional human RS1 packaged in the novel AIM™ capsid AAV204, has shown...