Deliver with confidence

Crosses BBB reaching up to 50% of neurons in the NHP cortex at a dose of 3e13 vg/kg.

Exceptional liver detargeting in mouse and NHP, with >10x lower biodistribution than AAV9.

Best capsid when compared head-to-head with other ALPL-targeting capsids in mouse and NHP.

Engages human Alkaline Phosphatase (ALPL) receptor for BBB-crossing, supporting translation to humans.

Available for large-scale manufacturing, ensuring clinical comparability and predictability.

Crosses BBB reaching up to 93% of neurons in humanized TfR1 mice at a dose of 2e13 vg/kg, with 113x higher brain transduction vs AAV9.

Exceptional liver detargeting in both mice and NHPs, with 29x lower biodistribution than AAV9.

Outperforms BI-hTFR1 and BI-hTFR1-v2 in brain transduction in humanized TfR1 mice.

Engages highly conserved human transferrin receptor (hTfR) receptor for BBB-crossing, supporting translation to humans.

Available for large-scale manufacturing, ensuring clinical comparability and predictability.

Crosses BBB reaching up to 30% of NHP neurons 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 NHPs.

Even at the low dose of 5e12 vg/kg (25x lower dose than Elevidys), transduces 73-88% of myofibers in NHP skeletal muscles.

Safe and broad muscle delivery, due to low dose and low biodistribution to liver and spleen.

Transduces 35-42% of NHP cardiac cells at 1.6e13 vg/kg.

Confidence in human translation given highly conserved entry receptor and in vitro transduction of human muscle cell lines.

Easily manufactured at a large scale and compatible with existing AAV9-based processes.

AAVrh74-based, with stronger muscle transduction compared to other natural AAV serotypes.

Even at the low dose of 5e12 vg/kg (25x lower dose than Elevidys), transduces 74% of myofibers in NHP skeletal muscles.

Safe and broad muscle delivery, due to low dose and low biodistribution to liver (1.4x < AAVrh74).

Confidence in human translation given in vitro transduction of human muscle cell lines.

Easily manufactured at a large scale and compatible with existing AAV9-based processes.

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.

Confidence in human translation given highly conserved entry receptor and in vitro transduction of human muscle cell lines.

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

>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.