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 4 Strategic Therapeutic Areas (STArs): genetic medicines, cardio-metabolic diseases, hepatic infectious diseases, and central nervous system (CNS) and ocular diseases.

  • LATE STAGE (PHASE 3)

    • Givosiran
      Acute Hepatic Porphyria

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

       

      Givosiran 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) by our collaborators at Sanofi Genzyme.

       

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

    • 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.

    • Lumasiran
      Primary Hyperoxaluria Type 1

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

       

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

    • Vutrisiran
      ATTR Amyloidosis

      Vutrisiran (ALN-TTRsc02) is a subcutaneously administered, investigational RNAi therapeutic targeting transthyretin (TTR) in development for the treatment of transthyretin-mediated (ATTR) amyloidosis.

       

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

  • EARLY STAGE (IND OR CTA FILED—PHASE 2)

    • Cemdisiran
      Complement-Mediated Diseases

      Cemdisiran (ALN-CC5) is a subcutaneously administered, investigational RNAi therapeutic targeting the C5 component of the complement pathway in development for the treatment of complement-mediated diseases.

       

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

    • ALN-AAT02
      ALPHA-1 LIVER DISEASE

      ALN-AAT02 is a subcutaneously administered, investigational RNAi therapeutic targeting alpha-1 antitrypsin (AAT) in development for the treatment of AAT deficiency-associated liver disease (alpha-1 liver disease).

       

      ALN-AAT02 utilizes our Enhanced Stabilization Chemistry-Plus (ESC+)-GalNAc delivery platform.

    • ALN-HBV02
      CHRONIC HEPATITIS B VIRUS INFECTION

      ALN-HBV02 is a subcutaneously administered, investigational RNAi therapeutic targeting the hepatitis B virus (HBV) genome for the treatment of chronic HBV infection by our collaborators at Vir Biotechnology. ALN-HBV02 is designed to inhibit expression of all HBV proteins, including hepatitis B surface antigen (HBsAg).

       

      ALN-HBV02 utilizes our Enhanced Stabilization Chemistry-Plus (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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