Abeona Therapeutics announces update on AAV ophthalmology program

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The company is advancing AAV-based gene therapy candidates toward IND studies on encouraging animal proof of concept data in Stargardt disease, X-linked retinoschisis and autosomal dominant optic atrophy.

Abeona Therapeutics’ preclinical programs are investigating the use of novel adeno-associated virus (AAV) capsids in therapies for serious genetic eye diseases. (Image courtesy of Adobe Stock/lightpoet)

Abeona Therapeutics’ preclinical programs are investigating the use of novel adeno-associated virus (AAV) capsids in therapies for serious genetic eye diseases. (Image courtesy of Adobe Stock/lightpoet)

Abeona Therapeutics Inc. this week announced three internally developed investigational preclinical gene therapy product candidates from its ophthalmology program.

According to the company, its 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,” Brian Kevany, PhD, chief technical officer and head of Research at Abeona, said in a news release. “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.”

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.

ABO-503 for X-linked retinoschisis

ABO-503, composed of a functional human RS1 packaged in the novel AIM capsid AAV204, has shown preclinical efficacy following delivery to the retina in a mouse model of XLRS. Preclinical studies have demonstrated robust RS1 expression in the retina, improved cone photoreceptor density and overall photoreceptor cell survival, as well as a restoration of outer retina architecture.

Preclinical Product Candidate ABO-505 for Autosomal Dominant Optic Atrophy (ADOA)

ABO-505 is designed to express a functional copy of human Opa1 in the retina following para-retinal injection. AB0-505 aims to take advantage of the robust optic nerve and retinal ganglion cell (RGC) transduction ability of AAV204 to deliver its genetic payload to the cells most affected by ADOA. Preclinical studies have confirmed expression of Opa1 in both cell culture and the retinas of dosed wild-type and disease model animals. Initial efficacy results suggest an improvement in retinal signaling to the brain, and improved visual acuity in treated mutant mice.

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