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Dicerna's RNAi Technology Shows Encouraging Action Against CNS Targets In Animal Studies


Dicerna Pharmaceuticals Inc (NASDAQ: DRNA) has announced new data from preclinical studies of its GalXC-Plus RNAi technology demonstrating its potential to deliver messenger RNA (mRNA) knockdown against prespecified gene targets across the central nervous system (CNS) and to specific CNS cell types.

  • Data from a preclinical mouse study showed that a single dose of an unconjugated GalXC-Plus molecule produced by the ALDH2 gene resulted in dose-dependent reductions of up to 92% knockdown in target mRNA and lasted through the trial conclusion at 28 days.
  • GalXC-Plus delivered similar mRNA reductions in non-human primate (NHP) studies after a single dose, resulting in up to 90% target mRNA silencing after 28 days. There were no adverse observations for any GalXC-Plus cohort in these trials.
  • Additional preclinical data demonstrated the degree and distribution of GalXC-Plus silencing of β-tubulin III (TUBB3 gene, expressed in neurons and associated with various cancers) and two undisclosed gene targets expressed by astrocytes and oligodendrocytes, respectively, using unconjugated and various conjugated GalXC-Plus payloads.
  • Price Action: DRNA shares are up 4% at $24.18 in the market trading session on the last check Tuesday.

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Posted-In: gene therapy mRNABiotech News Health Care Small Cap General

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