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Research programme: small interfering RNA-based therapeutics - Alnylam Pharmaceuticals

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Drug Profile

Research programme: small interfering RNA-based therapeutics - Alnylam Pharmaceuticals

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

Latest Information Update: 31 Mar 2022

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At a glance

  • Originator Alnylam Pharmaceuticals
  • Developer Alnylam Pharmaceuticals; Sanofi; Technical University Munich; University of Heidelberg
  • Class Anti-infectives; Antianaemics; Antihaemorrhagics; Antihyperlipidaemics; Hepatoprotectants; Small interfering RNA; Urologics
  • Mechanism of Action ANGPTL3 protein inhibitors; Antithrombin III inhibitors; Factor XII inhibitors; Glycolate oxidase expression inhibitors; Hepcidin inhibitors; Prealbumin inhibitors; Programmed cell death-1 ligand-1 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

Highest Development Phases

  • Suspended Anaemia
  • No development reported Adenovirus infections; Amyloidosis; Haemoglobinopathies; Haemophilia; Hepatitis; Hepatitis B; Hepatitis D; Hereditary angioedema; Hyperlipoproteinaemia type V; Hypertriglyceridaemia; Inborn genetic disorders; Liver disorders; Metabolic disorders; Primary hyperoxaluria

Most Recent Events

  • 28 May 2021 No recent reports of development identified for preclinical development in Hepatitis-B(Combination therapy) in Germany (SC)
  • 28 Apr 2020 No recent reports of development identified for preclinical development in Hereditary-angioedema in USA (Parenteral)
  • 06 Jan 2020 The Medicines Company has been acquired by Novartis
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