- Lysosomal storage diseases, including Gaucher Disease, are orphan drug designated diseases
- Lysosomal storage diseases are a group of approximately 50 rare inherited metabolic disorders that are characterized by an abnormal build-up of various toxic materials in the body's cells as a result of enzyme deficiencies
- Gaucher Disease and other lysosomal storage diseases originate from genetic mutations that result in “misfolded” or mutated enzymes
- Even though these enzymes are misfolded many, including Glucocerebrosidase (the enzyme deficiency responsible for Gaucher Disease), are functional
- Misfolded enzymes are naturally destroyed via Endoplasmic Reticulum Associated Degradation (ERAD), which results in the disease state caused by critical enzyme deficiencies
ERAD Therapeutics Inc.
The website is temporarily under construction and will be available soon.
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WHO WE ARE
- Private, pre-clinical gene therapy and orphan drug company
- Experienced Senior Management team and Board of Directors.
- On-going collaborations with Key stakeholders in Range Genetic Diseases.
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WHAT WE DO
- With an initial focus on neuromuscular and lysosomal storage diseases, we develop novel gene therapies to treat rare genetic diseases.
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OUR APPROACH
- We are taking a methodical approach to targeted gene therapies as well as a novel gene therapy-based approach to a broad spectrum of rare genetic diseases.
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WHY OUR CANDIDATES
- Few if any therapies exist to address the neurological aspects of rare genetic diseases.
- Our candidates are capable of being delivered across the blood brain barrier.
- Our therapeutic platforms will potentially address a multitude of rare genetic diseases.
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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.