Our

 Pipeline

Alnylam is leading the translation of RNAi (RNA interference) into a whole new class of innovative medicines to potentially address the needs of patients who have limited or inadequate treatment options. Our pipeline of investigational RNAi therapeutics is focused on diseases with high unmet medical need that fall under 3 Strategic Therapeutic Areas (STArs): genetic medicines, cardio-metabolic diseases, and hepatic infectious diseases.

pipeline chart

  • LATE STAGE (PHASE 3)

    • Patisiran
      Hereditary ATTR Amyloidosis

      Patisiran is an investigational RNAi therapeutic targeting transthyretin (TTR) in development for the treatment of hereditary ATTR amyloidosis (hATTR amyloidosis).

       

      Patisiran is the only investigational medicine in our pipeline that utilizes a lipid nanoparticle (LNP) formulation and is administered via intravenous infusion.

    • Inclisiran
      Hypercholesterolemia

      Inclisiran (ALN-PCSsc) is a subcutaneously administered, investigational RNAi therapeutic targeting proprotein convertase subtilisin kexin type 9 (PCSK9) in development for the treatment of hypercholesterolemia by our collaborators at The Medicines Company.

       

      Inclisiran utilizes our Enhanced Stabilization Chemistry (ESC)-GalNAc delivery platform.

    • Fitusiran
      Hemophilia and Rare Bleeding Disorders

      Fitusiran (ALN-AT3) is a subcutaneously administered, investigational RNAi therapeutic targeting antithrombin (AT) in development for the treatment of hemophilia and rare bleeding disorders (RBDs).

       

      Fitusiran utilizes our Enhanced Stabilization Chemistry (ESC)-GalNAc delivery platform.

    • Givosiran
      Acute Hepatic Porphyrias

      Givosiran (ALN-AS1) is a subcutaneously administered, investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyrias (AHPs), including acute intermittent porphyria (AIP), variegate porphyria (VP), and hereditary coproporphyria (HCP).

       

      Givosiran utilizes our Enhanced Stabilization Chemistry (ESC)-GalNAc delivery platform.

  • EARLY STAGE (IND FILED—PHASE 2)

    • ALN-CC5
      Complement-Mediated Diseases

      ALN-CC5 is a subcutaneously administered, investigational RNAi therapeutic targeting complement component C5 in development for the treatment of complement-mediated disease.

       

      ALN-CC5 utilizes our Enhanced Stabilization Chemistry (ESC)-GalNAc delivery platform.

    • ALN-GO1
      Primary Hyperoxaluria Type 1

      ALN-GO1 is a subcutaneously administered, investigational RNAi therapeutic targeting glycolate oxidase (GO) in development for the treatment of Primary Hyperoxaluria Type 1 (PH1).

       

      ALN-GO1 utilizes our Enhanced Stabilization Chemistry (ESC)-GalNAc delivery platform.

    • ALN-TTRsc02
      Hereditary ATTR Amyloidosis

      ALN-TTRsc02 is a subcutaneously administered, investigational RNAi therapeutic targeting the mRNA that codes for transthyretin (TTR) for the treatment of ATTR amyloidosis.

       

      ALN-TTRsc02 utilizes our Enhanced Stabilization Chemistry (ESC)-GalNAc delivery platform.

    • ALN-HBV
      Chronic Hepatitis B Virus Infection

      ALN-HBV is a subcutaneously administered, investigational RNAi therapeutic targeting the hepatitis B virus (HBV) gene for the treatment of chronic HBV infection.

       

      ALN-HBV utilizes our Enhanced Stabilization Chemistry (ESC)-GalNAc delivery platform.

Learn more about the latest in Alnylam news from Capella—the destination for updates on our progress in translating the science of RNAi to innovative potential medicines.

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