Erad Therapeutics Inc.

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.

The Orphan Drug Act (ODA), a federal law established in 1983, aims to encourage pharmaceutical companies to develop drugs for rare diseases with small markets

Focused on rare diseases affecting less than 200,000 patients in the U.S.
Over 55 million people are estimated to suffer from a rare disease in the U.S. and Europe
Global estimates are between 5,000 and 7,000 rare diseases
ODA provides multiple financial incentives for orphan drug development
Although the patient population is small orphan drugs have a higher per patient cost, acting as an incentive to develop drugs for orphan diseases
Since 1983, more than 400 orphan products have received U.S. market authorization
Japan and Europe have enacted similar legislation

MISSION STATEMENT

INNOVATIVE SCIENCE

WHO WE ARE

WHAT WE DO

OUR APPROACH

WHY OUR CANDIDATES

NEUROMUSCULAR DISEASE

GAUCHER DISEASE

Orphan Drug Overview