Research programme: small interfering RNA-based therapeutics - Alnylam

Drug Profile

Research programme: small interfering RNA-based therapeutics - Alnylam

Alternative Names: 5 x 15™ programme - Alnylam; ALAS-1 siRNA; ALN ANG; ALN F12; ALN-AAT02; ALN-AC3; ALN-AGT; ALN-APC; ALN-HDV; ALN-HPN; ALN-PDL; ALN-TMP; DLin-MC3-DMA LNP; ESC-GalNAc-Conjugate-HBV

Latest Information Update: 28 Feb 2017

Price : $50

At a glance

  • Originator Alnylam Pharmaceuticals
  • Developer Alnylam Pharmaceuticals; Charite of Berlin; Sanofi Genzyme
  • Class Small interfering RNA
  • Mechanism of Action Alpha 1 antitrypsin inhibitors; Angiotensinogen inhibitors; ANGPTL3 protein inhibitors; Antithrombin III inhibitors; CD274 antigen inhibitors; Factor XII inhibitors; Glycolate oxidase expression inhibitors; Hepcidin inhibitors; Prealbumin inhibitors; RNA interference; TMPRSS6 protein inhibitors
  • Orphan Drug Status

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

    Yes - Haemophilia A; Primary hyperoxaluria; Haemophilia B
  • New Molecular Entity Yes
  • Available For Licensing Yes

Highest Development Phases

  • Preclinical Adenovirus infections; Alpha 1-antitrypsin deficiency; Amyloidosis; Haemoglobinopathies; Haemophilia; Hereditary angioedema; Hyperlipoproteinaemia type V; Hypertriglyceridaemia; Preeclampsia; Primary hyperoxaluria
  • Research Hepatitis; Hepatitis D; Inborn genetic disorders; Liver disorders; Metabolic disorders
  • Suspended Anaemia

Most Recent Events

  • 08 Feb 2017 The Supreme Court of British Columbia grants a pre-trial injunction filed against Acuitas Therapeutics by Arbutus Biopharma for Arbutus's lipid nanoparticle (LNP) technology
  • 28 Sep 2016 Alnylam Pharmaceuticals announces intention to submit clinical trial application (CTA) for Alpha 1-antitrypsin deficiency in 2017
  • 04 Mar 2016 Preclinical trials in Hereditary angioedema in USA (Parenteral) (before March 2016; 3176776)
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