Immortalized human myoblast cell lines for the delivery of therapeutic proteins using encapsulated cell technology

Mol Ther Methods Clin Dev. 2022 Aug 1;26:441-458. doi: 10.1016/j.omtm.2022.07.017. eCollection 2022 Sep 8.

Abstract

Despite many promising results obtained in previous preclinical studies, the clinical development of encapsulated cell technology (ECT) for the delivery of therapeutic proteins from macrocapsules is still limited, mainly due to the lack of an allogeneic cell line compatible with therapeutic application in humans. In our work, we generated an immortalized human myoblast cell line specifically tailored for macroencapsulation. In the present report, we characterized the immortalized myoblasts and described the engineering process required for the delivery of functional therapeutic proteins including a cytokine, monoclonal antibodies and a viral antigen. We observed that, when encapsulated, the novel myoblast cell line can be efficiently frozen, stored, and thawed, which limits the challenge imposed by the manufacture and supply of encapsulated cell-based therapeutic products. Our results suggest that this versatile allogeneic cell line represents the next step toward a broader development and therapeutic use of ECT.

Keywords: allogeneic cell transplant; cell engineering; cell therapy; drug delivery; encapsulated cell technology; ex vivo gene therapy; macroencapsulation; myoblast.