RARE GENETIC DISEASES
THE UNMET NEED
Rare genetic diseases affect 5-10% of the global population - that's at least 350 million people.1
These conditions cause horrific suffering and often, early death.
Treatment options for rare genetic diseases are extremely limited, focusing on symptom management, rather than cure.
THE PIONEERING SOLUTION
The new technology developed by ChiraGen allows mutant genes to be selectively ‘switched off’. The treatment uses a new type of short sequence synthetic nucleotides. These bind up faulty genes so that they are not expressed and cannot cause disease.
Initially aimed at conditions such as Huntington’s disease, the treatment has potential for a wide range of rare genetic diseases.
Sharp Edge Labs
Biopharmaceuticals addressing rare genetic diseases, specifically a subset of mutations found in many genetic diseases associated with protein trafficking disorders.
1. RARE Diseases: Facts and Statistics https://globalgenes.org/rare-diseases-facts-statistics Accessed April 2018.