Intravitreal origin
The vector is administered into the vitreous in preclinical studies, a route familiar in ophthalmology practice.
Retinal penetrating AAV
AAV‑IKV is designed to move from the vitreous to the outer retina.
The platform was developed to address a delivery problem in ocular gene therapy: transgenes intended for photoreceptors or RPE often require access to cells beyond the inner retina.
Delivery problem
Outer retinal cells are important targets but difficult intravitreal targets.
Photoreceptors and RPE are central to retinal degeneration, macular degeneration, and inherited retinal disease. AAV‑IKV is being developed to improve access to these cells from the vitreous.
Retinal penetration
The design objective is movement across inner retinal barriers rather than expression limited to inner retinal cell populations.
Outer retinal expression
Published murine data support expression in photoreceptors and RPE following intravitreal administration.
Capsid and cell-surface biology
A receptor-informed design strategy.
AAV‑IKV grew from work on retinal cell surface interactions, Nuc1-mediated macromolecule uptake, and capsid engineering. The platform uses a peptide sequence incorporated into an AAV capsid context to alter retinal access.
VectorAAV‑IKV
Retinal penetrating AAV platform for transgene delivery.
RouteIntravitreal
Delivery from the vitreous toward outer retinal targets.
TargetsPhotoreceptors and RPE
Cell types central to retinal degeneration and AMD biology.
Program read-through
The same delivery logic supports multiple ocular programs.
Decorin delivery
Testing AAV‑IKV mediated decorin expression for CNV, fibrosis, inflammation, and autophagy readouts.
Complement targeting
Undisclosed complement-focused program for dry AMD and geographic atrophy biology.
Anterior segment delivery
Intracameral AAV‑IKV studies extend the platform to trabecular meshwork and outflow biology.
Complementary modality
Nuc1 extends the delivery thesis beyond AAV.
Nuc1 is a cell‑penetrating chaperone designed to support delivery of proteins, antibodies, peptides, steroids, and large molecular cargo into retinal cells without chemical conjugation to the cargo.
Protein delivery
Potential intracellular delivery of recombinant proteins and biologics.
AAV enhancement
Preclinical studies evaluate Nuc1 as a delivery enhancer for large cargo, including AAV.
Payload breadth
The approach supports payload classes that are not well suited to standard extracellular biologic strategies.