Alnylam Pharmaceuticals Clinical Trial Pipeline — 24 Active Studies

Alnylam Pharmaceuticals pipeline at a glance (March 2026)

About the Alnylam Pharmaceuticals clinical program

Alnylam Pharmaceuticals pioneered RNA interference (RNAi) as a therapeutic modality and has translated that platform into five approved medicines and one of the most productive rare disease pipelines in the industry. Their GalNAc-conjugated siRNA platform enables precise, durable gene silencing in the liver — enabling twice-yearly or even once-yearly dosing for previously untreatable conditions. Amvuttra (vutrisiran) and Onpattro (patisiran) have transformed the treatment of hereditary transthyretin-mediated amyloidosis (hATTR) — a fatal rare disease with limited options. Leqvio (inclisiran), partnered with Novartis, extends RNAi into mainstream cardiovascular disease with twice-yearly PCSK9 silencing for LDL reduction. Beyond approved medicines, Alnylam is systematically expanding into ATTR cardiomyopathy (a much larger population than hATTR polyneuropathy), hypertension (zilebesiran targeting angiotensinogen), and metabolic disease including obesity (ALN-2232). Their pipeline also includes programs in Huntington's disease (ALN-HTT02) and central nervous system applications.

Key therapeutic areas

• ATTR amyloidosis (hereditary and wild-type, polyneuropathy and cardiomyopathy)
• Cardiovascular disease (PCSK9, angiotensinogen, hypertension)
• Rare liver disease (acute hepatic porphyria, primary hyperoxaluria)
• Metabolic disease (obesity, diabetes)
• Neurodegenerative disease (Huntington's)
• Complement-mediated diseases

Key pipeline programs

• Amvuttra (vutrisiran) — siRNA, hATTR-PN + ATTR-CM (cardiomyopathy)
• Onpattro (patisiran) — first-approved siRNA, hATTR-PN
• Leqvio (inclisiran, Novartis) — PCSK9 siRNA, LDL reduction
• Givlaari (givosiran) — ALAS1 siRNA, acute hepatic porphyria
• Zilebesiran (ALN-AGT) — angiotensinogen siRNA, hypertension
• ALN-2232 — siRNA for obesity (undisclosed target)

Top Alnylam Pharmaceuticals trial indications

Condition / Indication	Trials
["Transthyretin Amyloidosis (ATTR) With Cardiomyopathy"]	3
["Transthyretin Amyloidosis With Cardiomyopathy"]	2
["Cerebral Amyloid Angiopathy"]	1
["Acute Hepatic Porphyria"]	1
["Transthyretin-Mediated Amyloidosis"]	1
["Advanced Hepatocellular Carcinoma", "Metastatic Hepatocellular Carcinoma"]	1
["Huntington's Disease"]	1
["Amyloidosis, Hereditary", "Transthyretin Amyloidosis"]	1

Why monitor Alnylam Pharmaceuticals's clinical trial activity

Alnylam's expansion of Amvuttra into ATTR cardiomyopathy is the most important rare disease label expansion of 2026 — the cardiomyopathy population is 10-50x larger than the polyneuropathy population it was originally approved in. Their zilebesiran hypertension program (partnered with Roche) could be the first RNAi drug in a mainstream CV indication. Each new Alnylam trial registration signals potential expansion of the RNAi modality into new therapeutic areas.

DataLookout monitors ClinicalTrials.gov daily and surfaces changes to Alnylam Pharmaceuticals's registered studies — new trial registrations, status transitions (e.g., "Not yet recruiting" → "Recruiting"), site additions, and protocol amendments. This is the intelligence layer that bridges the gap between quarterly investor calls and real-time pipeline developments.

How DataLookout tracks Alnylam Pharmaceuticals trials

• Sponsor normalization: Alnylam Pharmaceuticals may register trials under multiple legal entity names. DataLookout normalizes all related entities to a single canonical sponsor for complete pipeline coverage.
• Daily updates: ClinicalTrials.gov is queried each morning. New registrations and updates appear in that day's digest.
• Field-level change detection: Status changes, enrollment updates, and site additions are detected and surfaced — not just new registrations.
• Phase filtering: Focus on Phase 3 pivotal trials, Phase 2 proof-of-concept studies, or all phases for comprehensive coverage.
• Indication filtering: Combine sponsor monitoring with indication keywords to focus on the therapeutic areas relevant to your business.

Frequently asked questions

What is RNAi and why does it matter for Alnylam's pipeline?

RNA interference (RNAi) uses short interfering RNA (siRNA) to silently degrade specific messenger RNA (mRNA) sequences before they are translated into protein. Alnylam's GalNAc-conjugated siRNA platform delivers these molecules selectively to hepatocytes (liver cells) via GalNAc sugar-receptor targeting. Once inside the cell, a single siRNA dose can silence the target gene for 6-12 months. This enables twice-yearly or annual dosing for conditions like ATTR amyloidosis, hypertension, and hypercholesterolemia — a significant compliance and efficacy advantage over daily pills.

How many Alnylam trials are currently recruiting?

Based on current ClinicalTrials.gov data, 17 Alnylam-sponsored trials are actively recruiting. Alnylam's trial count is growing rapidly as they expand approved medicines into new indications (ATTR-CM, new cardiovascular applications) and advance next-generation siRNA programs.

Can DataLookout alert me to new Alnylam trial registrations?

Yes. DataLookout monitors ClinicalTrials.gov daily for all Alnylam-registered trial activity. Create a watchlist for Alnylam to receive alerts on new indication registrations, enrollment openings, and protocol amendments — particularly useful for tracking zilebesiran's cardiovascular expansion and the ALN-2232 obesity program.