DELIVERING RNAi-BASED BREAKTHROUGH THERAPIES

To Improve Lives

Our Pipeline

Dicerna is developing a pipeline of innovative RNAi therapies for the treatment of rare diseases, chronic liver diseases, cardiovascular disease, and viral liver infectious diseases based on our subcutaneously administered GalXCTM RNAi technology platform

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GalXC

The GalXCTM RNAi Technology Platform

GalXCTM is a proprietary RNAi technology platform that advances the development of next-generation RNAi-based therapies that act by silencing disease-driving genes in the liver. GalXC molecules are designed for infrequent subcutaneous administration.

GalXC Mechanism of Action

Unlocking the Potential of RNAi

RNA interference (RNAi) is a natural biological method for silencing or “turning off” specific genes known to cause or drive disease. In the RNAi process, small, carefully selected molecules of RNA inhibit the expression of these harmful genes by causing the targeted destruction of their messenger RNAs (mRNAs). RNAi has the potential to generate a revolution in medicine.on in medicine.

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Unlocking the potential of RNAi

RNAi: Timeline of Technological and Therapeutic Advances