ERAD Therapeutics Inc.
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WHO WE ARE
- Private pre-clinical orphan drug company
- Experienced Senior Management team and Board of Directors.
- Ongoing collaborations with the Hospital for Sick Children .
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WHAT WE DO
- With an initial focus on Tay-Sachs Disease, we create novel biological agents to treat mis-folded protein diseases (ERAD diseases)..
- We address the needs of patients with both systemic and neurological complications of their ERAD diseases.
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OUR APPROACH
- Our Principal Discovery gives us the ability to prevent destruction of mutant enzymes.
- This allows us to restore enzymatic activity to functional levels and thereby help reverse different disease states.
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WHY OUR DRUGS
- Our drug enters all cells in the body and can cross the blood brain barrier.
- No existing therapy addresses the neurological complications of ERAD diseases.
- We are creating a drug platform to potentially address multiple diseases.
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TAY-SACHS DISEASE
Tay-Sachs is a rare and usually fatal genetic disorder caused by the absence of-hexosaminidase (HexA). This missing enzyme causes progressive neurological deterioration. Symptoms include muscles weakness, twitching or jerking of muscles, lack of motor skills, and inability to make eye contact. Children with the most agressive form of Tay-Sachs Disease usually die by 3 to 5 years of age. A definitive treatment Tay-Sachs Disease does not yet exist.
There are 3 forms of Tay-Sachs Diseases determined by the age when symptoms first appear:
Classic Infantile Tay-Sachs -Symptoms appear around 6 months of age
Juvenile Tay-Sachs -Symptoms typically appear between ages 2 and 5, but can occur anytime during childhood.
Late Onset Tay-Sachs -Symptoms typically appear in adolescence or early adulthood, but can appear later.
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.