KU体育 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 | ???? |
| AMVUTTRA? (vutrisiran) | ATTR Amyloidosis Cardiomyopathy (CM) | COMMERCIAL | ???? |
| Nucresiran (ALN-TTRsc04) | ATTR Amyloidosis | PHASE 1 | ? |
PRODUCT
DISEASE
PHASE 1
PHASE 2
PHASE 3
COMMERCIAL
| GIVLAARI? (givosiran) | Acute Hepatic Porphyria (AHP) | COMMERCIAL | ???? |
| OXLUMO? (lumasiran) | Primary Hyperoxaluria Type 1 (PH1) | COMMERCIAL | ???? |
| Qfitlia? (fitusiran) | Hemophilia1 | COMMERCIAL | ???? |
| Cemdisiran (+/- Pozelimab) | Myasthenia Gravis1 | PHASE 3 | ??? |
| Cemdisiran (+Pozelimab) | Paroxysmal Nocturnal Hemoglobinuria1 | PHASE 3 | ??? |
| ALN-6400 | Bleeding Disorders | PHASE 1 | ? |
PRODUCT
DISEASE
PHASE 1
PHASE 2
PHASE 3
COMMERCIAL
| Leqvio? (inclisiran) | Hypercholesterolemia1 | COMMERCIAL | ???? |
| Zilebesiran | Hypertension2 | PHASE 2 | ?? |
| Zilebesiran + REVERSIR | Hypertension2 | PHASE 1 | ? |
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 | ? |
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, KU体育-led development with US profit split and milestones/royalties ex-US
3 Partner-led with profit split
4 Partnered, KU体育-led with profit split
5 Partner-led with KU体育 option for profit-split
KU体育 clinical development pipeline updated as of March 2025
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 KU体育-sponsored clinical trials and find one near you.
Capella
Learn more about the latest in KU体育 news from Capella—the destination for updates on our progress in translating the science of RNAi to innovative potential medicines.
Investors
KU体育 has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Discover more information for investors.