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Abeona Therapeutics Announces New Preclinical Data from its AAV Ophthalmology Program at the 26th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)
Animal proof-of-concept for AAV-based gene therapies for Stargardt disease, X-linked retinoschisis and autosomal dominant optic atrophy CLEVELAND, May 16,
About this update from Abeona Therapeutics Inc.
[{"type":"text","content":"Animal proof-of-concept for AAV-based gene therapies for Stargardt disease, X-linked retinoschisis and autosomal dominant optic atrophy\nCLEVELAND, May 16, 2023 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced new data regarding three internally developed preclinical gene therapy product candidates from its adeno-associated virus (AAV) ophthalmology program that will be presented at the 26th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), taking place from May 16-20, 2023 in Los Angeles, CA. “The preclinical proof-of-concept data being presented at ASGCT provides early evidence of the potential of our proprietary AAV capsids and gene constructs to express the recombinant protein in target tissues and rescue mutant phenotypes in mouse disease models,” said Brian Kevany, Ph.D., Chief Technical Officer and Head of Research at Abeona. “We look forward to pre-Investigational New Drug Application meetings with the FDA for two of our programs to take place in the second quarter of 2023.” Details for the poster presentations are as follows. Wednesday, May 17, 2023 at 12:00 PM PT In the presentation, entitled, “In Vivo Production of Full-Length ABCA4 Protein Following Cre-Mediated Recombination from Dual AAV Vectors in ABCA4-/- Mice,” Dr. Paul Wille, Director, Product Development, Abeona Therapeutics will present key findings: A dual AAV vector strategy using Cre recombinase efficiently reconstituted the ABCA4 gene, leading to full-length hABCA4 protein expression both in vitro via viral transduction of HEK293 cells and in vivo in photoreceptor cells after subretinal injection into ABCA4-/- mouse eyes.Cre-mediated recombination of ABCA4 was confirmed by mRNA sequencing of RNA from transfected HEK293 cells and AAV-dosed animals.Immunohistochemistry results confirmed correct localization of recombinant hABCA4 protein in photoreceptor cells. Thursday, May 18, 2023 at 12:00 PM PT In the presentation, entitled, “ABO-503, a Novel Gene Therapy for Treatment of X-Linked Retinoschisis,” Dr. Joseph Fogerty, Senior Scientist, Product Development, Abeona Therapeutics will present key findings: Robust RS1 expression was observed in photoreceptor cells near the injection site and in the adjacent inner retina in mutant mice six months after treatment with ABO-503.RS1 expression was associated with impro...