Delivery, delivery, delivery are the often cited biggest problems associated with unlocking the transformative potential of gene therapies. Genetic medicine approaches such as mRNA, gene silencing, gene editing and DNA gene therapies have curative potential for a host of disease indications previously untreatable. They have yet to enter the mainstream, due to safety concerns, complex manufacturing & difficulties delivering them efficiently to areas other than the liver, kidney & circulatory system.
The OmniSome platform piggybacks on the natural abilities of stem cell extracellular vesicles to efficiently deliver RNA cargoes from cell to cell and addresses difficulties related to safe & effective gene transfer via traditional approaches using viral vectors (e.g., lentiviruses & AAVs) and non-viral vectors (e.g., liposome and lipid, protein, and polymeric nanoparticles). Stem cell EVs have regenerative medicinal properties and are highly advantageous compared to other EVs based carriers (e.g., algae/milk derived EVs and HEK293 cells).
Competing viral and non-viral gene transfer vectors are inefficient & can cause inflammation, immunogenicity & elicit poor functional gene transfer.
OmniSomes lung targeting technologies offer an exemplar delivery platform for treating respiratory diseases and are the focus of the company’s therapeutic pipeline. The first problem is that gene transfer vectors must reach the lung via aerosol delivery. Trapping of the vectors in viscous mucus in the lung is a 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 these 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 gene transfer (see images below). 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. OmniSomes have a unique capacity to regenerate and repair the functional capacity of the diseased damaged lung, repair is essential to improve lung function which becomes severely impaired in people with chronic inflammatory lung diseases. The stem cells used in research and product development are ethically sourced from healthy, consenting donors (bone-marrow or adipose tissues).