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Genea Biocells develops therapeutic interventions for neuromuscular diseases – rare diseases affecting some part of the neuromuscular system such as skeletal muscles, peripheral motor neurons (in arms, legs, neck and face) or motor neurons in the spinal cord, and 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 the age of 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 current programs are:

Indication     Stage of Development    
  Discovery   IND-enabling   Phase 1/2
FSHD type 1
GBCC0905
FSHD type 2
GBCC0905
SMA
 
Kennedy's disease (SBMA)
 
LAMA2 (MDC1A)
 
Congenital Myotonic Dystrophy
 

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 Biocells’ 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.