CAMBRIDGE -
New data demonstrate the potential for the Company's
'We were pleased to share a robust data package highlighting the transformative capacity of our non-viral ARMMs technology to overcome fundamental delivery challenges that limit the potential of promising new treatment modalities,' said Paulash Mohsen, Chief Executive Officer at Vesigen. 'Based on the substantial evidence generated to date, we believe our approach enables highly efficient, tunable, and re-dosable delivery of multiple emerging therapeutic modalities. As we advance toward the clinic, we continue to evaluate partnerships where we may combine our
In
Data presentations demonstrated engineering of ARMMs to package and functionally deliver CRISPR/Cas9 complexes, base editing complexes, messenger RNA (mRNA), and short hairpin RNA (shRNA) to a diverse range of cell types in vitro and in vivo.
In vivo, a single intranasal administration of ARMMs loaded with gene or base editors targeting the immune modulatory genes NLRP3 or IRF5 resulted in 60+% on-target editing in macrophages and blunted the release of associated cytokines in murine lung tissue.
In vitro evaluation of Vesigen-engineered ARMMs loaded with CRISPR/Cas9 or base editors, in collaboration with
In Vivo Biodistribution of Engineered ARMMs
Two data presentations highlighted in vivo biodistribution of engineered ARMMs in non-human primates and rodents. In mouse models, functional delivery of engineered ARMMs was observed in the retina, lung, spleen, liver, and other tissues. In non-human primates, data showed delivery of engineered ARMMs intrathecally to cells in the peripheral nervous system.
Advances in
Three data presentations, including collaborations with
About Vesigen Therapeutics
Vesigen Therapeutics is a biotechnology company developing a novel, non-viral delivery technology for gene editing, RNA, and protein-based therapeutics. Vesigen's patented technology, called ARMMs (ARRDC1 Mediated Microvesicles), can be used to precisely deliver a wide range of payloads to a unique set of tissue and cell types. Vesigen has demonstrated highly efficient in vitro and in vivo functional delivery of a range of payloads across multiple cell types and is committed to developing transformative medicines that address current unmet medical needs. ARMMs were discovered and engineered into a drug delivery system at the
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