Open AccessRegulatory considerations for developing a phase I investigational new drug application for autologous induced pluripotent stem cells ‐based therapy product
Author(s) -
Jha Balendu Shekhar,
Farnoodian Mitra,
Bharti Kapil
Publication year - 2021
Publication title -
stem cells translational medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.781
H-Index - 71
eISSN - 2157-6580
pISSN - 2157-6564
DOI - 10.1002/sctm.20-0242
Subject(s) - induced pluripotent stem cell , reprogramming , regenerative medicine , medicine , drug development , stem cell , good manufacturing practice , cell therapy , clinical trial , bioinformatics , microbiology and biotechnology , drug , pharmacology , biology , embryonic stem cell , cell , biochemistry , genetics , gene , regulatory affairs
Induced pluripotent stem cells (iPSC)‐based therapies have been hailed as the future of regenerative medicine because of their potential to provide treatment options for most degenerative diseases. A key promise of iPSC‐based therapies is the possibility of an autologous transplant that may engraft better in the longer‐term due to its compatibility with the patient's immune system. Despite over a decade of research, clinical translation of autologous iPSC‐based therapies has been slow—partly due to a lacking pre‐defined regulatory path. Here, we outline regulatory considerations for developing an autologous iPSC‐based product and challenges associated with the clinical manufacturing of autologous iPSCs and their derivatives. These challenges include donor tissue source, reprogramming methods, heterogeneity of differentiated cells, controls for the manufacturing process, and preclinical considerations. A robust manufacturing process with appropriate quality controls and well‐informed, prospectively designed preclinical studies provide a path toward successful approval of autologous iPSC‐based therapies.