An automated platform for the large-scale production of miniaturized neuromuscular organoids

Introduction

Organoids have been developed as advanced 3D cell culture systems that resemble aspects of the in vivo tissues and provide an alternative to study the mechanisms of human disease and identify novel treatments.

Recent Developments

The last years have witnessed tremendous developments in the field of stem cell and organoid research, but the full potential of these systems remains to be exploited.

Challenges in Organoid Research

Two major challenges facing the organoid field are:

1. Reproducibility
2. Scalability

The manual production of organoids is a labor-intensive and expensive process. The development of cost-effective, fast, and reliable methods is a prerequisite for transferring organoid technologies to the industry for high-throughput approaches.

Novel NMO Model

We have recently established a novel complex human neuromuscular organoid (NMO) model from human pluripotent stem cell-derived neuromesodermal progenitors. NMOs self-organize into spinal cord neurons and skeletal muscle compartments that contract by forming functional neuromuscular junctions.

ERC Consolidator Grant: GPSorganoids

The ERC consolidator grant “GPSorganoids” focuses on:

• The generation of position-specific (GPS) NMOs representing distinct spinal cord segments
• The use of such NMO models to study the selective vulnerability of specific spinal cord neurons to neuromuscular diseases like amyotrophic lateral sclerosis and spinal muscular atrophy.

PoC Grant and Commercialization

The PoC grant goes beyond the scope of our ERC consolidator grant and focuses on the commercialization of the NMO model through the establishment of an automated, reliable, and high-throughput production line that could apply to industry settings.

Ultimate Goal

Our ultimate goal is to establish NMOs as a leading model in the market for high-throughput drug screening approaches and accelerate the development of novel therapies for neuromuscular disorders.