Deliver with confidence

>40x retinal transduction vs AAV2, outperforms all tested externally engineered capsids at doses of 1e10-1e11 vg/eye.

An ideal capsid for optogenetic gene therapies: 5x transduction of bipolar cells in the inner nuclear layer compared to a reference externally engineered capsid.

Intravitreal injection enables easy administration and broad patient access.

Optimized and validated in NHPs and mice, supporting translation to humans.

Easily manufactured at large scales and compatible with AAV2-based processes

80x higher retinal transduction vs AAV2, with similar production efficiency

Transduces retinal ganglion cells, bipolar cells and rod photoreceptors

Reaches macula and peripheral retina, enabling biofactory, neuroprotective, optogenetic and photoreceptor therapies

Easy intravitreal injection enables broad patient access

Optimized and validated in NHPs

Transduces 50-90% of myofibers in NHP skeletal muscles, and multiple regions of the heart and CNS at doses as low as 4e12 vg/kg.

Low biodistribution to off-target peripheral organs, with liver biodistribution <10 vg/dg and spleen <0.5 vg/dg at a dose of 4e12 vg/kg, supporting safer IV dosing.

Intravenous injection enables more uniform transduction across cells and tissues and broader patient access.

Identification of highly conserved candidate receptor supports translation to humans

Easily manufactured at large scales and compatible with AAV9-based processes.

Crosses BBB reaching up to 30% of NHP neurons pan-brain at a dose of 3e13 vg/kg, with 280x higher brain transduction vs AAV9.

50x lower liver transduction compared to AAV9.

Intravenous injection enables more uniform brain transduction and broader patient access.

Optimized and validated in NHPs.

Highly conserved candidate receptor for BBB-crossing supports translation to humans.

100x higher brain transduction and 10x lower liver transduction vs. AAV9, with similar production efficiency

Transduces neurons and glia, including cell-types relevant for ALS, Huntington’s, Parkinson’s Disease

Reaches deep brain regions and the spinal cord

Easy intravenous injection enables uniform transduction and broad patient access

Optimized and validated in NHP