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 Oct 2018

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At a glance

  • Originator Alnylam Pharmaceuticals
  • Developer Alnylam Pharmaceuticals; Charite - Universitatsmedizin Berlin; Sanofi Genzyme; Technical University Munich; University of Heidelberg
  • Class Small interfering RNA
  • Mechanism of Action Alpha 1 antitrypsin inhibitors; Angiotensinogen inhibitors; 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 B; Haemophilia A; Primary hyperoxaluria
  • New Molecular Entity Yes
  • Available For Licensing Yes

Highest Development Phases

  • Preclinical Alpha 1-antitrypsin deficiency; Hepatitis B; Hereditary angioedema; Primary hyperoxaluria
  • Research Hepatitis; Inborn genetic disorders; Metabolic disorders
  • Suspended Anaemia
  • No development reported Adenovirus infections; Amyloidosis; Haemoglobinopathies; Haemophilia; Hepatitis D; Hyperlipoproteinaemia type V; Hypertriglyceridaemia; Liver disorders; Preeclampsia

Most Recent Events

  • 28 Oct 2018 No recent reports of development identified for preclinical development in Preeclampsia in Germany (SC)
  • 03 Oct 2018 Alnylam Pharmaceuticals submits a CTA to the Medicines and Healthcare products Regulatory Agency for a phase I/II trial in Alpha-1 antitrypsin deficiency
  • 03 Oct 2018 Alnylam Pharmaceuticals plans a phase I/II trial for ALN AAT 02 in Apha-1 antitrypsin deficiency patients and healthy volunteers in United Kingdom by the end of 2018
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