Originally Aired: August 15, 2022
Time: 8:00 am PT, 11:00 am ET, 17:00 CET
In this Learning Lab, we discussed the development of respiratory airway and alveolar organoids derived from human iPSCs and how these are used in modeling respiratory diseases such as cystic fibrosis. Airway organoid models can be used for testing the therapeutic efficacy of candidate drugs for respiratory diseases. Regenerated airway and alveolar cells derived from human iPSCs express proteins that match human respiratory cell biomarkers and play an important role in pathogenesis, such as ACE2 receptor protein in SARS-CoV-2 infections. Our panelists will discuss scaling, standardization, gene editing, co-culture and imaging of these organoid disease models, as important resources for pharmacologic, toxicologic, functional and efficacy studies in respiratory drug development initiatives.
Clinical development of drugs for respiratory diseases such as COVID-19, idiopathic pulmonary fibrosis, cystic fibrosis, pneumonia, COPD and lung cancers has met major obstacles, particularly during phase 2, when therapeutic efficacy the candidate drug is typically first assessed. Pluripotent stem cells have the innate potential for unrestricted self-renewal and the ability to differentiate into any cell type in the body. However, isolating these cells from live animals is challenging. In 2012 Shinya Yamanaka and Sir John Gurdon were awarded the Nobel Prize for directly generating induced pluripotent stem cells (iPSCs) from somatic cells by introducing four genes, now collectively called the Yamanaka factors (Myc, Oct3/4, Sox2 and Klf4). Regenerative medicine aims to derive specialized cell types from iPSCs that can provide disease models for therapeutic development and can be used to transplant, replace, engineer or regenerate diseased cells.
A live Q&A session followed the presentation, offering a chance to pose questions to our expert panelists.