To Improve Lives

Our Pipeline

Dicerna is developing a pipeline of innovative RNAi therapies using our GalXCTM RNAi platform for the treatment of diseases involving the liver, including rare diseases, viral infectious diseases, chronic liver diseases and cardiovascular diseases.

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Global Genes RARE Cast with Daniel Levine

Dr. Ralf Rosskamp, Dicerna’s chief medical officer, participated in podcast with RARECast and provided an overview of the challenges facing drug development in rare diseases, as well as an update on Dicerna’s development program in primary hyperoxaluria.

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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.

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Unlocking the potential of RNAi

RNAi: Timeline of Technological and Therapeutic Advances