Our Science

OUR PIPELINE

Alnylam has led the translation of RNAi (RNA interference) into an innovative new class of medicines which silence the genes that cause disease or that contribute to disease. Our pioneering science and pipeline of investigational RNAi therapeutics are focused on addressing the needs of patients who have limited or inadequate treatment options for diseases that currently fall under 4 Strategic Therapeutic Areas (STArs): genetic medicines, cardio-metabolic diseases, infectious diseases, and central nervous system (CNS) and ocular diseases.

Our RNAi therapeutics platform and drug development approach aims to generate 2-4 new investigational new drug (IND) applications each year.

Learn more about our clinical trials here ›

Alnylam Clinical Development Pipeline

pipeline

Late Stage (Phase 3)

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 Novartis.

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

Lumasiran

SEVERE Primary Hyperoxauluria type 1

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

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

Early Stage (IND or CTA Filed—Phase 2)

Cemdisiran

Complement - Mediated Disease

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.

Zilebesiran

Hypertension

Zilebesiran (ALN-AGT01) is a subcutaneously administered, investigational RNAi therapeutic targeting angiotensinogen (AGT) in development for the treatment of hypertension in high unmet need populations.

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

ALN-HSD

Non-Alcoholic Steatohepatitis

ALN-HSD is a subcutaneously administered, investigational RNAi therapeutic targeting HSD17B13 for the treatment of non-alcoholic steatohepatitis (NASH), in collaboration with Regeneron.

ALN-HSD 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.

Learn More ›

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