Researchers at Harvard University identified a new intercellular communication mechanism regulated by ARRDC1 (ARRestin Domain Containing Protein 1), which drives vesicle formation and budding. These unique vesicles, called ARMMs (ARRDC1-Mediated Microvesicles), transport signaling molecules between cells. Under an exclusive license from Harvard, Vesigen engineers ARMMs to efficiently deliver therapeutic payloads, including gene-editing complexes, RNA, and proteins.
Preclinical studies, including in non-human primates, have confirmed that our engineered ARMMs can be safely administered through varying routes of administration. We’ve shown that ARMMs can deliver a variety of payloads to a wide range of tissues—including retinal, lung, nervous system, liver, and spleen.
To create our therapeutic ARMMs, we engineer producer cells to express the ARRDC1 gene tethered to the payload of interest. ARRDC1 then generates ARMMs loaded with our cargo, which bud from the cell surface and can be easily collected and purified.
We are expanding the biodistribution of ARMMs by engineering surface-associated targeting moieties that can direct uptake in specific cell types. These include hematopoietic stem cells (HSCs), T cells, B cells, neurons and others. The ability to target ARMMs will allow for novel and previously unattainable in vivo therapeutic strategies for a large range of rare and complex diseases.
Vesigen Therapeutics
790 Memorial Drive
Suite 103
Cambridge, MA 02139
