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Alnylam Development Pipeline

Alnylam’s pipeline of investigational RNAi therapeutics is focused in 3 Strategic Therapeutic Areas (STArs):

  • Genetic Medicines, with a broad pipeline of RNAi therapeutics for the treatment of rare diseases;
  • Cardio-Metabolic Disease, with a pipeline of RNAi therapeutics toward genetically validated, liver-expressed disease targets for unmet needs in cardiovascular and metabolic diseases; and,
  • Hepatic Infectious Disease, with a pipeline of RNAi therapeutics that address the major global health challenges of hepatic infectious diseases.

Access to Investigational Drugs
Alnylam’s position on providing access to its investigational drugs prior to approval. Click here to learn more.

Development Programs
Signifies Human Proof of Concept
Discovery
Development
Phase 1
Phase 2
Phase 3
GENETIC MEDICINES
Patisiran
Fitusiran
ALN-CC5
ALN-AS1
ALN-GO1
ALN-TTRsc02
ALN-AATO2
ALN-TMP
ALN-F12
CARDIO-METABOLIC DISEASES
ALN-PCSsc
ALN-AC3
ALN-ANG
ALN-AGT
ALN-F12
HEPATIC INFECTIOUS DISEASES
ALN-HBV
ALN-HDV
ALN-PDL
Discovery
Development
Phase 1
Phase 2
Phase 3



GENETIC MEDICINES

Hereditary ATTR Amyloidosis
  - Patisiran (ALN-TTR02)
  - Revusiran (ALN-TTRsc)

Hemophilia and Rare Bleeding Disorders - ALN-AT3

Complement-Mediated Diseases - ALN-CC5

Hepatic Porphyrias - ALN-AS1

Alpha-1 Antitrypsin Deficiency - ALN-AAT

Primary Hyperoxaluria Type 1 - ALN-GO1

ATTR Amyloidosis - ALN-TTRsc02

Beta-Thalassemia / Iron Overload Disorders - ALN-TMP

Hereditary Angioedema - ALN-F12

Additional Genetic Medicine Programs

CARDIO-METABOLIC DISEASES

Hypercholesterolemia ALN-PCSsc

Mixed Hyperlipidemia and Hypertriglyceridemia - ALN-ANG

Hypertriglyceridemia - ALN-AC3

Mixed Hyperlipidemia / Hypertriglyceridemia - ALN-ANG

Hypertension / Preeclampsia - ALN-AGT

Thromboprophylaxis - ALN-F12

Additional Cardio-Metabolic Programs

HEPATIC INFECTIOUS DISEASES

Hepatitis B Virus Infection - ALN-HBV

Hepatitis D Virus Infection - ALN-HDV

Chronic Liver Infection - ALN-PDL

Additional Hepatic ID Programs