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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.

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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.

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Top Tier Pharmaceutical Companies are Active in Orphans

Top Tier Pharmaceutical Companiese are extremely active in the orphan drug space:

  • 40% of acquired biotechs between 2008-2012 had an orphan drug in development
  • Novartis, GSK, Roche and Pfizer are largest orphan drug companies
  • Pfizer, Gilead, Roche, Shire, BMY and Celgene are leading orphan drug acquirer
  • 50% of top 20 orphan drugs were either acquired or in-licensed by Top Tier Pharmaceutical Companies

Key Market Statistics:

  • Worldwide orphan drug sales forecast to grow to $178 billion by 2020 with a CAGR of +11.7% (2015 to 2020); almost double overall prescription market growth
  • Orphan drugs set to be 20% of worldwide prescription sales by 2020 (excluding generics)