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Next-generation GeneToBe Pipeline:
Liver Diseases, Usher Syndrome, Cystic Fibrosis

Metabolic dysfunction-associated steatohepatitis (siRNA)

Alcoholic liver disease (siRNA)

Usher syndrome (CRISPR)

Cystic fibrosis (CRISPR)

Cystic fibrosis (undisclosed*)

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Early discovery

Late discovery

IND-enabling

Development

*opportunity for indication expansion

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