NMJCare

Challenge

Neuromuscular diseases are highly prevalent (~1 in 3,500) and have no cure. Current preclinical models fail to capture human specificity and complexity. This results in costly false-positive drug candidates, late-stage failures, and high R&D costs.

Solution

NMJCare is a patented 2.5D iPSC-based physiologically relevant human neuromuscular system that combines the scalability of 2D with the physiological relevance of 3D models for high-throughput disease-relevant drug screening.

• Multicellular neuromuscular junction model (NMJ) generating neurons, muscle, and Schwann cells in one system
• Functionally active, capturing nerve-induced muscle contraction and disease-specific impairments (e.g. Spinal Muscular Atrophy, Duchenne Muscular Dystrophy, Myasthenia Gravis, Amyotrophic Lateral Sclerosis)
• Scalable, pharma-compatible 2.5D cultures enabling long-term, functional and morphological readouts

Development status

• TRL3‑4
• Faithfully models Spinal Muscular Atrophy (SMA) with clear functional and structural deficits
• First proof of concept achieved in SMA, demonstrated therapeutic rescue in pilot drug-response screens

Applications:

• Preclinical drug discovery and screening
• Patient-derived personalized screening and disease modeling
• Mechanism of action, toxicity and efficacy testing

IP

Self-organizing neuro-muscular junction cell culture
Prov. Applications (06÷2023): US, SG, CA

Publications

Efficient generation of a self-organizing neuromuscular junction model from human pluripotent stem cells
Urzi et al. (2023) Nat. Commun

Spin-Off

NMJCare is also a prefounded Spin-Off.

Lab

Gouti Lab

Stem Cell Modeling of Development and Disease 

News and background