Semi-automated optogenetic platform for evaluation of human neuromuscular functionality

This technology is a platform that uses patient-derived cells to accurately evaluate their neuromuscular junction function to detect and diagnose neuromuscular diseases such as myasthenia gravis (MG).

Unmet Need: Precision diagnostic tool for myasthenia gravis and other muscular disorders

Disorders in neuromuscular transmission are routinely diagnosed based on blood testing for biomarkers and electrodiagnosis tests. However, existing biomarkers often correlate poorly with disease severity, and electrodiagnosis is a painful process with readouts that often do not lead to a specific diagnosis. Furthermore, it remains challenging to evaluate a patient’s serum for its ability to inhibit neuromuscular activity and diagnose neuromuscular dysfunction in MG. As such, there remains a need for a robust, non-invasive method of evaluating neuromuscular functionality for improved diagnostics and monitoring of MG and other neuromuscular disorders.

The Technology: In vitro optogenetic system for evaluating neuromuscular function

This technology is a platform for in vitro testing of human neuromuscular functionality. It consists of a functional optogenetic model of the neuromuscular junction that analyzes optically sensitive motor neurons and contractile muscles, driven by a computer-controlled optical system. This enables simultaneous optical stimulation of motor neurons and detection of muscle contraction in response to stimulation. The technology further includes image processing software capable of generating a trace of contractile activity to determine the effectiveness of the formed neuromuscular junction. The platform can additionally be used to expose the neuromuscular junction to patient serum for accurate diagnoses of MG.

Applications:

• Diagnostics and evaluation of myasthenia gravis
• Diagnosis and evaluation for other neuromuscular diseases and disorders of the neuromuscular junction
• Research platform for the study of neuromuscular junction functionality
• System for drug screening

Advantages:

• Semi-automated system
• Requires only a small amount of patient serum
• Minimal user intervention
• Enables MG evaluation in “seronegative” patients
• Allows for direct determination of neuromuscular junction functionality
• Able to detect changes in neuromuscular transmission before visible patient symptoms develop

Lead Inventor:

Gordana Vunjak-Novakovic, Ph.D.

Patent Information:

Patent Pending (US 20210179989)

Related Publications:

• Vila OF, Uzel SG, Ma SP, Williams D, Pak J, Kamm RD, Vunjak-Novakovic G. “Quantification of human neuromuscular function through optogenetics” Theranostics. 2019 Jan 31; 9(5): 1232-1246.

• Vila OF, Qu Y and Vunjak-Novakovic G. “In vitro models of neuromuscular junctions and their potential for novel drug discovery and development” Expert Opinion in Drug Delivery. 2020 Mar; 15(3): 307-317.

• Vila OF, Chavez M, Ma SP, Yeager K, Zholudev LV, Colon-Mercadoc JM, Carmen Laib C, Feliciano C, Carter M, Judge L, Conklin B, Ward ME, McDevitt T, Vunjak-Novakovic G. “Bioengineered optogenetic model of human neuromuscular junction” Biomaterials Epub. 2021 Jul 30.

Tech Ventures Reference:

• IR CU18400

• Licensing Contact: Beth Kauderer