IQ MPS 

In 2023, the IQ MPS Affiliate collaborated with the U.S. Food and Drug Administration (FDA) to organize a virtual workshop to discuss the potential benefits of animal-cell based MPS in drug discovery and development, such the ability to bridge between animal in vivo data and human MPS results and other opportunities to advance 3Rs initiatives. Presentations given by IQ MPS, FDA, and NC3Rs (UK) representatives highlighted potential use cases for animal cell-based MPS and important considerations for their application. Breakout discussions provided an opportunity for other stakeholders to weigh in, including representatives from NIH/NCATS, NIHS, NIST, NIEHS/ICEATM, EURL ECVAM, Danish Medicines Agency, European Commission, Health Canada, and PMDA. Workshop organizers and presenters collaborated after the event to prepare a proceedings manuscript. READ HERE!

CRISPR-Cas9 gene editing technology offers promising potential for treating genetic diseases by correcting mutations. However, its safe application faces challenges due to limitations in testing models. Traditional laboratory methods using immortalized cells or animal studies often fail to accurately reflect human responses. Microphysiological systems (MPS), also known as "organs-on-chips," represent an innovative alternative that can better mimic human tissue structure and function. This review explores how combining CRISPR technology with MPS can enhance both fields: MPS provides more relevant testing platforms for CRISPR therapies, while CRISPR enables creation of disease models in MPS. This synergy could lead to safer, more effective gene therapies and reduce the need for animal testing. By offering more accurate prediction of human responses, this approach addresses current limitations in translating laboratory findings to clinical applications, ultimately benefiting patients with genetic disorders. READ HERE!

This paper reviews considerations and recommendations for adopting animal MPS models in drug discovery and development and describes how their deployment is consistent with 3Rs principles. Animal MPS availability would fill a key gap in the ability to examine in vitro to in vivo translatability. Since in vivo animal data will be available and guide decision making in regulatory activities for the foreseeable future, there is significant opportunity for translational assessments. In vivo animal study findings that are recapitulated using in vitro models generated from the corresponding animal species provide validation that those models possess the relevant and necessary attributes, e.g., species-specific pharmacodynamics, metabolism, transport, susceptibility to toxicity, etc. Results from the corresponding human models can then be interpreted with greater confidence for the relevant context of use (COU). Read HERE.