IntraStem

IntraStem is a new non-viral intracellular delivery system being applied for local applications

It has a wide range of potential applications from gene therapy, gene silencing through to mRNA delivery. 

At present, biomedical gene therapy approaches generally utilise viral vectors to ’transfect’ the target cell with the intended genetic material e.g. gene. However, they have a number of limitations with restrict their applications (safety, tolerability, limited delivery options and they are very expensive and complex to manufacture) while non-viral transfection approaches such as electroporation cannot be used in vivo and also have other key limitations (cell toxicity and limited scale).

IntraStem utilises combinations of novel peptide sequences to achieve major gains in transfection efficiency – both potency and viability. It can deliver a wide range of biologic molecules or ‘cargoes’ including nucleic acids (plasmid (p)DNA, mRNA and siRNA), enzymes, transcription factors, antibodies and gene editing complexes such as CRISPR/Cas9 into cells without the limitations seen with existing systems. Importantly, IntraStem exhibits low cell toxicity and so cells can be exposed without directly affecting their viability, presenting opportunities for both ex vivo and in vivo therapeutic applications. These key advantages are also supported by the opportunity for low cost and simple manufacture, enabling a potential step change in pricing or profitability from current gene therapy products.

IntraStem therefore has a broad range of potential applications including gene therapy in vivo and ex vivo cell therapy modification. 

Locate is currently evaluating a number of new product opportunities for in-house development and potential new third party collaborations.