High Risk Cardiovascular Disease; Hypertension; High Cardiovascular Risk
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Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
Status note Subcutaneously administered RNAi therapeutic; company pursuing regulatory approval for cardiomyopathy indication in additional jurisdictions outside the US.
ONPATTRO
Approved · ActiveModality: siRNA
Aliases patisiran
Target TTR
Indications
hATTR amyloidosis with polyneuropathy(Approved)
Status note Intravenously administered RNAi therapeutic.
GIVLAARI
Approved · ActiveModality: siRNA
Aliases givosiran
Target ALAS1
Indications
acute hepatic porphyria(Approved)
OXLUMO
Approved · ActiveModality: siRNA
Aliases lumasiran
Target HAO1
Indications
primary hyperoxaluria type 1(Approved)
Leqvio
Approved · ActiveModality: siRNA
Aliases inclisiran
Target PCSK9
Partnerships Novartis (commercialized by Novartis; ex-US rights via MDCO license)
Indications
hypercholesterolemia(Approved)
Status note July 2025 FDA label update enabled monotherapy use with diet and exercise to reduce LDL-C in adults with hypercholesterolemia.
Qfitlia
Approved · ActiveModality: siRNA
Aliases fitusiran
Target AT (antithrombin)
Partnerships Sanofi (Genzyme; commercialized by Sanofi)
Indications
hemophilia A or B(Approved)
Status note FDA approved March 2025 for routine prophylaxis in patients 12+ with hemophilia A or B with or without inhibitors; NMPA approved December 2025.
zilebesiran
Phase 3 · ActiveModality: siRNA
Target angiotensinogen (AGT)
Partnerships Roche (worldwide strategic collaboration for joint development; Alnylam received $675M license obligation transaction price)
Indications
hypertension(Phase 3)
Status note Investigational siRNA targeting liver-expressed angiotensinogen, in Phase 3 for hypertension.
mivelsiran
Stage not disclosed · ActiveModality: siRNA
Aliases ALN-APP
Target amyloid precursor protein (APP)
Indications
cerebral amyloid angiopathy (CAA)(Stage not disclosed)
Alzheimer's disease(Stage not disclosed)
Status note Intrathecally administered RNAi therapeutic; filing places it under early-stage clinical development programs; current phase not stated.
nucresiran
Phase 1 · ActiveModality: siRNA
Target TTR
Indications
ATTR amyloidosis(Phase 1)
Catalysts
— by 2028
— by 2030
Status note Next-generation TTR silencer using IKARIA technology; positive interim Phase 1 results announced November 2024 (rapid TTR knockdown sustained six months after a single dose).
cemdisiran
Phase 3 · ActiveModality: siRNA
Target C5
Partnerships Regeneron (solely responsible for development, manufacturing and commercialization; Alnylam receives milestones and tiered double-digit royalties)
Status note Regeneron advancing cemdisiran in combination with its anti-C5 antibody pozelimab in Phase 3 for PNH, and as monotherapy and in combination with pozelimab in Phase 3 for myasthenia gravis and geographic atrophy.
Per-asset detail extracted from the 10-Q filed 2026-07-30. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.
Pipeline
Lead drug
Nucresiran
Lead indication
Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy
Sponsor-matched active trials from ClinicalTrials.gov (26 as sponsor, 1 as collaborator); "Sponsor" = company is the lead sponsor, "Collaborator" = company is a collaborator. The BP column flags big-pharma involvement as lead sponsor or collaborator (excluding the company itself). The Mgmt guide column shows what management guided for readout timing when a guided program matches the trial (hover for the quote, click for the source). Trial listings are not a company pipeline slide.
Institutional holder positions from 13F filings. Q/Q change is vs the prior quarter's filing; % of portfolio is the fund's own reported portfolio weight.
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