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Alnylam Clinical Development Pipeline

TRANSTHYRETIN AMYLOIDOSIS (TTR)

PRODUCT

DISEASE

PHASE 1

PHASE 2

PHASE 3

COMMERCIAL

ONPATTRO® (patisiran) hATTR Amyloidosis Polyneuropathy (PN) COMMERCIAL     
AMVUTTRA® (vutrisiran) hATTR Amyloidosis Polyneuropathy (PN) COMMERCIAL     
Vutrisiran ATTR Amyloidosis Cardiomyopathy (CM) PHASE 3    
ALN-TTRsc04 ATTR Amyloidosis PHASE 1  
RARE

PRODUCT

DISEASE

PHASE 1

PHASE 2

PHASE 3

COMMERCIAL

GIVLAARI® (givosiran) Acute Hepatic Porphyria (AHP) COMMERCIAL     
OXLUMO® (lumasiran) Primary Hyperoxaluria Type 1 (PH1) COMMERCIAL     
Fitusiran Hemophilia1 PHASE 3    
Cemdisiran (+/- Pozelimab) Myasthenia Gravis1 PHASE 3    
Cemdisiran (+Pozelimab) Paroxysmal Nocturnal Hemoglobinuria1 PHASE 3    
CARDIOVASCULAR

PRODUCT

DISEASE

PHASE 1

PHASE 2

PHASE 3

COMMERCIAL

Leqvio® (inclisiran) Hypercholesterolemia1 COMMERCIAL     
Zilebesiran Hypertension2 PHASE 2   
METABOLIC

PRODUCT

DISEASE

PHASE 1

PHASE 2

PHASE 3

COMMERCIAL

ALN-HSD NASH1 PHASE 2   
ALN-PNP NASH3 PHASE 1  
NEUROLOGIC

PRODUCT

DISEASE

PHASE 1

PHASE 2

PHASE 3

COMMERCIAL

Mivelsiran (ALN-APP) Cerebral Amyloid Angiopathy (CAA) PHASE 2   
Mivelsiran (ALN-APP) Alzheimer’s Disease PHASE 1  
ALN-HTT02 Huntington’s Disease4 PHASE 1  
ALN-SOD SOD1 Amyotrophic Lateral Sclerosis (ALS)3 PHASE 1  
OTHER

PRODUCT

DISEASE

PHASE 1

PHASE 2

PHASE 3

COMMERCIAL

Cemdisiran (+/- Pozelimab) Geographic Atrophy1 PHASE 3    
Elebsiran (ALN-HBV02/VIR-2218) Hepatitis B Virus Infection5 PHASE 2   
Elebsiran (ALN-HBV02/VIR-2218) Hepatitis D Virus Infection5 PHASE 2   
ALN-BCAT Hepatocellular Carcinoma PHASE 1  
ALN-ANG3 Healthy Volunteers1 PHASE 1  

1 Out-licensed with milestones and/or royalties

2 Partnered, Alnylam-led development with US profit split and milestones/royalties ex-US

3 Partner-led with profit split

4 Partnered, Alnylam-led with profit split

5 Partner-led with Alnylam option for profit-split

Alnylam clinical development pipeline updated as of October 2024

Additional Information

Clinical Trials

Patients are at the center of everything we do and we work tirelessly to bring new medicines to those with unmet needs. Learn more about Alnylam-sponsored clinical trials and find one near you.

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Capella

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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Investors

Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Discover more information for investors.

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