Research programme: Dicer-substrate siRNA therapeutics - Dicerna

Drug Profile

Research programme: Dicer-substrate siRNA therapeutics - Dicerna

Alternative Names: CTNNB1 DsiRNA; DCR BCAT; DCR-HBV; DCR-PHXC; Dicer siRNA - Dicerna; DsiRNA - Dicerna; DsiRNA-EX conjugate - Dicerna; GalNAc-DsiRNA-EX conjugates; GalXC therapy

Latest Information Update: 04 Nov 2017

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At a glance

  • Originator Dicerna Pharmaceuticals
  • Class Antihyperglycaemics; Antihyperlipidaemics; Antineoplastics; Antirheumatics; Small interfering RNA
  • Mechanism of Action Glycolate oxidase expression inhibitors; RNA interference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Haematological malignancies; Hepatitis B; Hypercholesterolaemia; Primary hyperoxaluria
  • Research Liver disorders
  • No development reported Solid tumours
  • Discontinued Hepatitis C; Hyperlipidaemia; Rheumatoid arthritis; Type 2 diabetes mellitus

Most Recent Events

  • 04 Nov 2017 No recent reports of development identified for preclinical development in Solid-tumours in USA (Parenteral)
  • 02 Nov 2017 Dicerna pharmaceuticals plans to file an IND application with the US FDA in USA for Primary hyperoxaluria in the first quarter of 2018
  • 16 Oct 2017 Preclinical trials in Primary hyperoxaluria in United Kingdom (SC) before October 2017
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