RARE GENETIC DISEASES

THE UNMET NEED

 
 
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Rare genetic diseases affect 5-10% of the global population - that's at least 350 million people.1

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These conditions cause horrific suffering and often, early death.

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Treatment options for rare genetic diseases are extremely limited, focusing on symptom management, rather than cure.

 
 

 
 

THE PIONEERING SOLUTION

 
 

NeuBase Therapeutics


NeuBase is a platform company that is pioneering a novel type of therapy that can address dominant genetic diseases.

 

Sharp Edge Labs


Sharp Edge Labs has a proprietary platform that allows high-throughput screening for drugs that rescue protein trafficking mutations in the genome. Thousands of genetic disease result from such mutations. These disorders include the most common rare genetic disease - cyctic fibrosis.

The company has several drugs that it has identified from its platform that it is maturing through development programs.

 
 

References

1. RARE Diseases: Facts and Statistics https://globalgenes.org/rare-diseases-facts-statistics Accessed April 2018.