Research programme: Dicer-substrate siRNA therapeutics - Dicerna

Drug Profile

Research programme: Dicer-substrate siRNA therapeutics - Dicerna

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

Latest Information Update: 29 Nov 2016

Price : $50

At a glance

  • Originator Dicerna Pharmaceuticals
  • Class 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.

  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Haematological malignancies; Primary hyperoxaluria; Solid tumours
  • Research Hypercholesterolaemia; Liver disorders; Unspecified
  • Discontinued Hepatitis C; Hyperlipidaemia; Rheumatoid arthritis; Type 2 diabetes mellitus

Most Recent Events

  • 07 Nov 2016 Dicerna plans a clinical trial for Primary hyperoxaluria
  • 07 Nov 2016 Early research in Hypercholesterolaemia in USA (SC)
  • 07 Nov 2016 Early research in Undefined indication in USA
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