Large-scale iPSC production system aimed at clinical therapy funded by NIH

Written by Alexandra Thompson

Allele Biotechnology and Pharmaceuticals (San Diego, CA, USA) has been awarded a Phase I SBIR grant by the NIH’s NHLBI to support the development of a large-scale, clinical-grade induced pluripotent stem cell manufacturing system aimed at producing stem cell-derived cells and tissues for clinical application.

The ability to produce consistent, effective cell-based therapies at a low cost and to a large scale is a significant challenge that the cell therapy and stem cell fields need to overcome for therapies to reach their clinical and commercial potential. Much work is being carried out to try and remove this barrier, and the NIH’s NHLBI has provided Allele Biotechnology and Pharmaceuticals (Allele) with a Phase I SBIR grant to support their work in developing a large-scale manufacturing platform that could produce clinical-grade iPSC-based cells and tissues for therapeutic use at a low cost.

Allele is a private, San Diego (CA, USA)-based company developing technologies and products for drug discovery and cell therapy applications. As part of their aim of reducing the cost of large-scale cell therapy manufacting — mostly caused by the high cost of clinical-grade growth factors and cytokines — the company previously developed a patented method of reprogramming somatic cells into iPSCs, secured industrial licensees using this technology and initiated cGMP procedures for clinical applications.

“We have been diligently working on removing the use of protein factors through our own proprietary protocols to generate many clinically-relevant cell types,” explained Dr Jiwu Wang, Allele’s CEO and the Principle Investigator of the new NIH grant. “By developing a recombinant protein-independent, real-time adjusted culture system under this project, we are confident that — as many groundbreaking technologies such as genome sequencing have done — the manufacturing process will mature and the costs will come down to eventually benefit everybody.”

The technology was submitted to the NIH and received a high score and funding. With the funding from the NIH scientific review panel, Allele’s researchers could help bring about a process for the production then differentiation of clinical-grade iPSCs into specific cell types for therapeutic application at a scale large enough to satisfy the clinical demand, which would be a great leap forward for the field. Moreover, as the technology uses iPSC cells, these therapeutic cells would have the added benefit of being immune matched to patients.