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About Us

With our leading gene therapy capabilities and highly credentialed scientific and management team, we play an essential role in the scientific community by advancing novel treatment options for treating human diseases.

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Pipeline

Our in-house programs target chronic and life-threatening conditions which are gene-associated, and for which there is no effective treatment or cure.

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Our Science

Permanent gene silencing with DNA-Directed RNA Interference (ddRNAi).

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