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Genea Biocells uses its proprietary stem cell technology platform for early discovery research to find diverse therapeutic interventions for neuromuscular diseases. Our current programs are:

Facioscapulohumeral muscular dystrophy (FSHD)

Adult onset muscle weakness affecting approx. 1:8,000

Caused by aberrant upregulation of the embryonic transcription factor DUX4

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Spinal muscular atrophy (SMA)

Leading genetic cause of childhood death, affecting approx. 1:11,000 children

Motor neurons and skeletal muscle contribute to disease

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Neuromuscular diseases are rare diseases affecting some part of the neuromuscular system such as:

Skeletal muscles

Peripheral motor nerves (in arms, legs, neck and face) or motor neurons in the spinal cord

Neuromuscular junctions, the connections between neurons and muscles

It is estimated that more than a million people in the United States are affected by some form of neuromuscular disease which are either acquired or genetic conditions. About 40 percent of patients are under age 18. Neuromuscular diseases are progressive in nature resulting in muscle weakness and fatigue. The onset of symptoms varies widely, as some diseases are present at birth, in childhood or in adults. Life expectancy also differs by disease and severity, from very short to normal length. Secondary effects of muscle deterioration such as heart and respiratory problems are often the cause of death.

Our Mission

At Genea Biocells we are committed to accelerating the development of therapies for neuromuscular diseases to make a positive impact in the lives of patients and their families.


Genea Biocell’s drug discovery efforts continue to benefit from our ongoing interactions with disease foundations and patient groups as we gain better a understanding of diseases and discover new therapies.