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MISSION STATEMENT

To improve the lives of patients suffering from rare genetic diseases (“RGD or Orphan Diseases”), and the lives of their families, friends and colleagues. Most patients suffering from Orphan Diseases typically have few, if any, treatment options. Our mission is to bring new biotherapeutics that can improve the lives of these patients via a robust research and development program.

OUR SCIENCE

 

ERAD Therapeutics Inc.

The website is temporarily under construction and will be available soon.

 

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INNOVATIVE SCIENCE

Delivering Protein Therapeutics to the CNS

NEUROMUSCULAR DISEASE

Neuromuscular diseases are a large group of disorders that primarily disrupt the normal functioning of muscles and the nerves that control them. This leads to muscle weakness, impaired movement, and often progressive disability.

Examples of neuromuscular diseases are:

  • Muscular Dystrophy including Duchenne, Becker and many more;
  • Charcot- Marie-Tooth Disease and other neuropathies;
  • Myesthenia-gravis and more

GAUCHER DISEASE

Gaucher Disease is the most common Lysosomal Storage Disease, yet it is still considered an Orphan Disease. Currently affecting over 8,000 American’s, it is a genetic disorder resulting in an enzyme deficiency. Type 1 disease usually results in an enlarged liver, enlarged spleen, anemia and bone disease. Without therapy this significantly impacts both the quality and duration of life. Type 3 disease impacts all of the above as well as causing progressive and ultimately fatal neurological deterioration.

Gaucher Disease Facts

Expired

There are ~8,000 total U.S. cases with a predominance in Ashkenazi Jews. Gaucher Disease is classified into 3 types according to neurological deterioration, age and progression rate:

Type 1: Most common; does not impact the Central Nervous System, and usually presents in adulthood with an enlarged liver, enlarged spleen, anemia, and bone disease

  • 7,000 U.S. patients
  • 5 therapies on the market with $1.6 billion in sales per year

Type 2: Exceedingly rare; involves the CNS, affects infants and is uniformly fatal by 2 years of age

  • ~250 U.S. patients
  • There are no therapies for Type 2 Gaucher Disease

Type 3: Begins at any time in childhood, involves the CNS, and is characterized by slowly progressive neurological deterioration and premature death

  • 750-1,000 U.S. patients
  • There are no therapies for Type 3 Gaucher Disease