Drug intelligence / Profile preview

AAV2.7m8

Development stage
Unknown
Lead developer
Adverum Biotechnologies
Modality
AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intravitreal, Intracochlear
01

Overview

AAV2.7m8 is an engineered adeno-associated virus (AAV) capsid variant developed through directed evolution to enhance gene delivery efficiency to retinal and cochlear cells. It is based on the AAV2 serotype with a specific 10-amino acid insertion in each of the triple spikes of the capsid, discovered via a “7-mer” library screen[2][4][6]. This modification enables robust transduction of both inner and outer hair cells in the cochlea as well as multiple layers of retinal cells following intravitreal injection[1][3][4]. In preclinical models, it has shown high efficiency for gene transfer in auditory and visual systems, making it valuable for inner ear gene therapy targeting hereditary hearing loss and for ocular gene therapy targeting diseases such as neovascular (wet) age-related macular degeneration (AMD), diabetic macular edema (DME), and retinitis pigmentosa[1][4][5][6]. In clinical trials, it serves as the vector backbone for investigational therapies like ADVM-022 (aflibercept expression cassette)[4][5][7]. While highly effective at transducing target tissues, administration can stimulate immune responses including intraocular inflammation due to its tendency to package heterogeneous genomes[6][8].

02

Targets

HSPG (Basement membrane-specific heparan sulfate proteoglycan core protein (perlecan))

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