Breakthrough in Personalized Cell Therapies for Compromised Patient Populations

Locate Therapeutics, a leading UK regenerative medicine company has announced that it has received a Biomedical Catalyst Primer Award for a potentially breakthrough personalized cell therapy development project. Called CellFuse, the project is designed to demonstrate the ability of its combined IntraStem synthetic peptide-based intracellular delivery system and TAOS^® polymer-based matrix to solve the current problem of low transfection efficiency in patients with already highly compromised cell populations.

Diabetes sufferers requiring spinal fusion has been chosen, because this group currently suffers poor clinical outcomes and more frequent complications after fusions with autograft or bone graft substitutes. Over 1 million fusions are performed annually in Europe and the US, with the numbers continuing to grow.

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The underlying causes of the failed outcomes and complications are firstly the low availability due to ageing of the mesenchymal stem cells (MSCs) required to generate new vascularized bone tissue and then their compromised function due to poor protection at administration sites. Studies show that up to 95% of cells are being lost using current methods. Based on modified Cell-Penetrating technology, IntraStem is able to address this by delivering immortalization RNAs into the patient’s cells in order to amplify their numbers, overcome proliferative shortfalls and expedite production of therapeutically-relevant numbers. TAOS^® then provides a robust delivery matrix that maintains cell viability and ensures differentiation and localization.

“Whilst growing, with over 600 trials underway worldwide, the Cell Therapy market is currently facing a major challenge – how to reduce cell death and increase therapeutic efficacy. IntraStem enables us to re-program cells to achieve beneficial outcomes. This system has been shown to increase intracellular delivery of protein by two-orders of magnitude and is especially effective at delivering these payloads to pluripotent stem cells that have proved resistant to conventional cell penetrating peptide strategies. TAOS^® then creates an ideal environment for the cells to differentiate,” says Dr. Robin Quirk, CEO of Locate Therapeutics. “We are therefore delighted to receive this grant which will enable us to further demonstrate the potential of our approach by taking a truly personalized cell therapy into clinic. Going forward we will also be looking to promote IntraStem’s potentially unrivalled efficiency for delivering antibodies, magnetic nanoparticles (MNPs) and nucleic acids (plasmid (p)DNA, mRNA and siRNA) into cells at much higher levels than the current alternatives for reprogramming, as well as improving gene editing, using for example, the CRISPR-cas9 approach.”

Locate plans to take the CellFuse product independently through market approval to commercialization. There is also the continued option of out-licensing or sale to a commercial development partner at the appropriate point in development. The award of £614, 952 amounts to 70% of the total £878, 503 project costs, with Locate Therapeutics contributing the remainder.