OmniSomes lung targeting technologies offer an exemplar delivery platform for treating respiratory diseases and are the initial focus of the company’s therapeutic pipeline (see below). The first problem to solve for pulmonary gene therapy is that the gene transfer vectors must efficiently reach the lung via aerosol delivery. Our partnership with Aerogen Limited has resulted in optimised OmniSome formulations with their state-of-the art vibrating mesh nebuliser technology – delivering OmniSomes with high efficiency while maintaining the structural integrity and functionality of the EVs.
Trapping of delivery vectors in viscous mucus in the lung is another significant barrier to gene transfer to the underlying target cells. Even if the mucus layer can be penetrated, inefficient penetration through the cell membrane further impedes access of traditional gene therapy vectors to the underlying target cells, thus preventing successful gene transfer. OmniSomes have exhibited mucus diffusion and tropism for cell entry and gene transfer in human lung cell models (air-liquid interface cell cultures) and have been successfully aerosolised in small animal studies to effect in vivo gene transfer.
Adding further complexity is the processing of the carriers inside the cells – EVs are naturally occurring carriers of RNA and other messengers from cell to cell in the body – this gene transfer, refined by millennia of evolution, has demonstrated 30-40 fold more efficient gene transfer than synthetic lipid nanoparticles*.
The benefits of the OmniSome technology are not just related to delivery and efficient gene transfer as they are derived from stem cells which have widely documented therapeutic efficacy – they are thus expected to further benefit patients afflicted by respiratory diseases through anti-inflammatory, anti-fibrotic, anti-microbial, immune cell modulatory and tissue regeneration effects. The stem cells used in research and product development are ethically sourced from healthy, consenting donors (bone-marrow or adipose tissues).
* Murphy DE, de Jong OG, Evers MJW, Nurazizah M, Schiffelers RM, Vader P. Natural or Synthetic RNA Delivery: A Stoichiometric Comparison of Extracellular Vesicles and Synthetic Nanoparticles. Nano Lett. 2021 Feb 24;21(4):1888-1895. doi: 10.1021/acs.nanolett.1c00094. Epub 2021 Feb 11. PMID: 33570966; PMCID: PMC8023702.
The properties of OmniSomes are applicable to a wide variety of diseases amenable to gene therapy, targeting other organs in the body (e.g., liver, CNS, digestive and cardiovascular systems).
Feel free to contact us to discuss how OmniSomes could enable the partnered co-development of your RNA therapeutic assets by leveraging our expertise in EV manufacturing and delivery technologies.