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DICERNA: Mechanism of action of GalXC RNAi therapy 

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Lipedema

medical illustration of This is a mechanism of action (MOA) illustration depicting GalXC RNAI therapy, a proprietary technology platform that advances the development of RNAi-based therapies to silence disease-causing genes in the liver. Each GalX molecule is a double-stranded RNA attached to GalNAc. GalXC binds to ASGPR receptors on target hepatocyte cells, enabling optimal delivery.
Next medical illustration of This mechanism of action illustration explains how the CRYSVITA molecule binds to FGF23 to normalize phosphorous levels in the bloodstream. CRYSVITA (burosumab-twza) is the only FDA-approved therapy targeting the underlying cause of XLH in adults and children 6 months or older. Client: Ultragenyx Pharmaceuticals

CRYSVITA Mechanism of Action

This is a mechanism of action (MOA) illustration depicting GalXC RNAI therapy, a proprietary technology platform that advances the development of RNAi-based therapies to silence disease-causing genes in the liver. Each GalX molecule is a double-stranded RNA attached to GalNAc. GalXC binds to ASGPR receptors on target hepatocyte cells, enabling optimal delivery.

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© Diana Kryski