iPSC France

The generation of human induced pluripotent stem cells (iPSCs) from adult cells offers a unique opportunity to obtain an unlimited supply of a broad spectrum of specialized cells, for innovative cell therapy strategies in the treatment of cancers, treatment degenerative diseases and as an alternative to organ transplants.

Producing an immuno-compatible or hypoimmunogenic iPSC cell population is an important challenge for promoting allogeneic cell therapies accessible to a large number of patients. iPS cells are particularly suitable for genome editing technology, which makes the development of “universal-donor” iPS lines a possible goal. The main advantage of an iPSC donor line derived from a single genetic background is the ease of preclinical testing, reducing expense and time, compared to the characterization of cell products derived from multiple donors and various iPS cell lines.

The project aims to develop optimal mRNA delivery methods to generate iPSC cells at reduced and controlled costs, preserving genetic integrity and reducing tumor risk. New strategic approaches will be developed to prevent allo-immunogenicity and “universal” iPSC candidates will be evaluated for their differentiation potential specifically in two lineages of clinical interest: the macrophages and neuronal cells. Two pathological models will be studied.

• Functional study of armed macrophages derived from human iPSC for the treatment of lung cancer
• Study of the survival and function of iPSC-derived neuron grafts for the treatment of Huntington’s disease (NH primate model).

IPS-France project’s partners bring strong complementary skills in bioengineering of iPSC cells, mRNA delivery and gene editing for the production in CiTHERA platform of a new generation of hypo-immunogenic iPSC in pharmaceutical grade suitable for the manufacture of “off-the-shelf” therapeutic products for future clinical programs.

The iPSC France project aims to develop a new generation of stem cells: cells that can transform into many different cell types. Produced from adult cells, these stem cells offer significant potential for treating diseases that are currently difficult to treat, including certain cancers and neurological disorders, and could also help reduce the need for organ transplantation.

One of the main challenges is to create cells that can be used in a large number of patients without being rejected by their immune systems. The project therefore aims to produce “universal” cells that are compatible with many recipients and can be prepared in advance, like a ready-to-use medicine.

The researchers will develop new manufacturing methods to improve the safety of these cells, reduce production costs, and minimize the risk associated with their use. They will then assess their potential in two applications: strengthening the immune system’s defenses against lung cancer and replacing nerve cells that are destroyed in Huntington’s disease.

By bringing together complementary expertise in cell biology and genetic engineering, the iPSC France project could help accelerate the development of innovative cell therapies that are safer, more affordable, and capable of benefiting a large number of patients.