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. LifeX Capital has invested into Sharp Edge Labs.

 
 

References

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