IntraStem is a cell penetrating peptide (CPP) based intracellular delivery system designed to overcome the limitations of current gene therapy vectors.
It raises the efficacy bar for non-viral transfection systems whilst offering greater versatility, expected safety and lower manufacturing costs compared with viral vectors.
As a next-generation approach, IntraStem is able to deliver a wide range of biologics including plasmid DNA, mRNA, siRNA, proteins and antibodies, with high efficiency in an array of cell types including many hard-to-transfect cells.
Repeated or sustained administration is also possible with IntraStem owing to its low toxicity profile, and good tissue penetration has been demonstrated following local delivery in vivo.
These combined attributes make IntraStem an ideal technology for the transfection of cells or more generally the delivery of biologics into cells in vivo and therefore enable cellular reprogramming or gene editing directly in patients.
Locate is currently evaluating new therapeutic applications for this exciting new technology.
Dr Nick Staples, Chief Executive of Locate, commented: "The granting of our first IntraStem patents in the US and Europe adds to the excitement building around our next generation, non viral transfection technology."
"We are delighted with the comparative performance of IntraStem versus current transfection standards, which has now extended across a wide range of cell types, payloads and preclinical models."
"The broad utility of IntraStem opens up a multitude of therapeutic opportunities, including gene editing and cell reprogramming in vivo. We are presently evaluating new product opportunities in therapy areas wherein cell and gene therapies may deliver breakthrough and potentially curative benefits to patients."