Science and Technology

Meeting the challenge of intracellular therapeutic targets

Current modalities have a limited ability to deliver certain types of therapeutic molecules to target cells. These technologies are hampered by toxicity, poor cell/tissue specificity, and reduced cellular activity due to endosomal trapping or endolysosomal processing. Vesigen’s novel therapeutic programs utilize ARMMs technology to package and safely deliver RNA, proteins, and gene-editing complexes to cells not accessible by existing delivery systems.

Extracellular vesicles and ARMMs technology

Vesigen’s technology is based on a distinct class of extracellular vesicles that are involved in signaling between cells and tissues in the body. Our ARMMs (ARrestin-domain 1 Mediated Microvesicles) technology is named for the cellular protein responsible for mediating vesicle formation: ARRestin Domain Containing Protein 1 (ARRDC1).

Analogy to enveloped viruses

In an analogous system, much of the cellular machinery used in the production of ARMMs is used by enveloped viruses to generate tropic and fusogenic viral particles. After infecting a target cell, enveloped viruses hijack cellular mechanisms to produce virus particles that are shed from the cell surface encapsulated by portions of the host cells’ plasma membrane. The Gag proteins of enveloped viruses mimic ARRDC1 in recruiting host cell proteins for ARMMs budding to generate viral particles with cell/tissue specificity and long serum half-life.

Vesigen’s founder discovered the inter-relationship of these systems and demonstrated the power and versatility of using ARMMs to turn a producer cell into a miniature, combined drug and formulation factory.

Universal solution for drug development

Engineered ARMMs provide a flexible platform for therapeutic delivery with the potential to overcome the limitations of existing technologies—including toxicity, low specificity, instability, and high immunogenicity associated with lipid nanoparticles (LNPs), viral vectors, transducing peptides, and nano-carriers.

Vesigen’s ARMMS system also offers important advantages over exosomes, LNPs, penetrating peptides, and viral delivery systems:

Broad tissue distribution observed in vivo

Production of payload and delivery technology in a single process

Scalable production system using common upstream and downstream biologics process technologies

Superior engineerability with applications to broad therapeutic classes

Meeting the challenge of intracellular therapeutic targets

Extracellular vesicles and ARMMs technology

Analogy to enveloped viruses

Universal solution for drug development

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