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Abeona Therapeutics announces update on AAV ophthalmology program


The company plans to advance 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.

medical equipment used for research and development sitting on a table

The company’s preclinical programs are investigating the use of novel AAV capsids in therapies for serious genetic eye diseases. (Image Credit: Adobe Stock/Shutter2U)

Abeona Therapeutics Inc. today announced 3 internally developed investigational preclinical gene therapy product candidates from its ophthalmology program.

According to a news release, the company’s preclinical programs are investigating the use of novel adeno-associated virus (AAV) capsids in therapies for serious genetic eye diseases.

Brian Kevany, PhD, chief technical officer and head of Research at Abeona, noted that the company is 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.

“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,” Kevany said in the news release. “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 FDA on the clinical development plans for these programs.”

ABO-504 for Stargardt Disease

Abeona noted that its 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

According to the company, 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.

The company noted that 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.

ABO-505 for Autosomal Dominant Optic Atrophy

Abeona noted in the news release 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.

The company noted 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.

Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Abeona’s lead clinical program is EB-101, its investigational autologous, engineered cell therapy currently in development for recessive dystrophic epidermolysis bullosa.

According to the release, Abeona is currently in the process of preparing a Biologics License Application (BLA) for submission to the FDA. The company’s development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need.

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