Ozmosi | AAV-204 Drug Profile
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AAV-204

Alternative Names: AAV-204, AAV204
Clinical Status: Active
Latest Update: 2025-10-13
Latest Update Note: News Article

Product Description

AAV204, a novel adeno-associated virus (AAV) capsid from Abeona's in-licensed AIM™ capsid library, has previously been shown to facilitate transduction of both the inner and outer retina after intravitreal administration in mice and non-human primates. The purpose of the current study was to evaluate in non-human primates transduction levels in the macula and optic nerve following administration of AAV204 directly into the vitreous of the eye by para-retinal administration, a recently-developed method, which unlike subretinal administration does not create a retinal detachment. AAV204.GFP or AAV8.GFP were administered to four non-human primate eyes and green fluorescent protein (GFP) expression was monitored using scanning laser ophthalmoscopy (SLO), followed by immunohistochemistry analysis at 28 days post-injection. (Sourced from: https://investors.abeonatherapeutics.com/press-releases/detail/232/abeona-therapeutics-announces-aav204-a-novel-aav-capsid)

Mechanisms of Action: Gene Therapy, GFP

Novel Mechanism: Yes

Modality: Gene Therapy

Route of Administration: N/A

FDA Designation: *

Approval Status: Not Approved

Approved Countries: None

Approved Indications: None

Known Adverse Events: None

Company: Abeona
Company Location: Eastern America
Company Founding Year: 2013
Additional Commercial Interests: None

Clinical Description

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Phase 0: Retinoschisis

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