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AAV204

Development stage: Preclinical
Lead developer: Abeona Therapeutics
Modality: Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration: Intravitreal, Para-retinal

Overview

AAV204 is a novel adeno-associated virus (AAV) capsid from the AIM capsid library, licensed by Abeona Therapeutics. It is designed for gene therapy, targeting the delivery of genetic payloads to tissues such as the retina, central nervous system, and others. AAV204 has shown high macular and optic nerve transduction levels after para-retinal administration, facilitating the transduction of both inner and outer retina after intravitreal administration in mice and non-human primates. It is being evaluated for potential gene therapies in ophthalmology indications.

Other names: AAV 204AAV204AAV-204ABO-401ABO401ABO 401

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