PROJECTS
AVAILABLE FOR PARTNERING
Goose
AAV-Goose is a rationally designed AAV library for efficient targeting of renal glomerular cells following IV delivery. In rodent models, IV-administered AAV-Goose shows robust and selective glomerular transduction, addressing the need for improved capsids to enable future gene therapies for kidney diseases where current vectors fall short.
Luzon
AAV-Luzon is engineered to efficiently target heart cells after IV injection. In non-human primates, the AAV-Luzon library achieves strong and widespread cardiac transduction, providing a powerful platform for developing gene therapies for cardiomyopathies, heart failure, and other cardiac indications.
Roadrunner
AAV-Roadrunner is designed for efficient skeletal muscle targeting via IV delivery. In non-human primates, IV-administered AAV-Roadrunner drives high-level expression across major muscle groups, supporting the development of systemic gene therapies for muscular dystrophies and other neuromuscular disorders.
Cassowary
AAV-Cassowary is designed for efficient pancreatic Langerhans islet targeting via IV delivery. In non-human primates, IV-administered AAV-Cassowary drives high-level expression across α and β cells, supporting the development of systemic gene therapies for pancreatic disorders.
Colibri
By integrating diffusion-based generative models and message-passing neural networks, this project enables end-to-end in silico design of receptor-targeting AAV capsid variant libraries. Computational candidates are ranked for receptor engagement and translatability, then experimentally screened in vitro and in vivo, for uptake and transduction, creating a tight design–test loop for rapid optimization.
Swift
AAV-Swift is a rationally engineered AAV vector family designed to maximize spread and transduction in the brain following intraparenchymal injection. In rodent brain studies, locally delivered AAV-Swift demonstrates markedly improved distribution and transgene expression compared with wild-type AAV2, achieving strong performance at up to 10,000× lower vector dose—supporting the development of efficient, focal gene delivery approaches across a broad range of indications.
PARTNERSHIPS
Kingfisher
rAAVen’s Kingfisher library is engineered to target oligodendrocytes with high precision, showing strong enrichment in white-matter cell populations. In collaboration with Myrtelle Inc., intraventricular screening identified five lead capsids that markedly outperform established oligodendrocyte-targeting benchmarks.
Bluejay
Bluejay is a collaboration with Prevail Therapeutics to develop novel AAV capsids for disorders of the nervous system. rAAVen leads capsid engineering, discovery, screening, and validation, while Prevail drives preclinical development, manufacturing, and clinical advancement to bring new CNS gene therapies to patients.