Alnylam Pharmaceuticals

What Alnylam Pharmaceuticals does

Alnylam Pharmaceuticals is a commercial-stage biopharmaceutical company founded in 2002 and headquartered in Cambridge, Massachusetts, that develops RNA interference (RNAi) therapeutics to selectively silence disease-causing genes. The company's core technology uses synthetic small interfering RNA (siRNA) molecules that bind to complementary messenger RNA (mRNA) and trigger catalytic degradation via the endogenous RNA-induced silencing complex (RISC). Alnylam operates three proprietary siRNA delivery platforms: Lipid Nanoparticles (LNP) for liver-targeted intravenous delivery, trivalent N-acetylgalactosamine (GalNAc) conjugates for subcutaneous liver delivery, and C16 conjugates for extrahepatic delivery to the central nervous system, lung, and eye. The company has secured six approved RNAi therapeutics: ONPATTRO (patisiran, 2018), GIVLAARI (givosiran, 2019), OXLUMO (lumasiran, 2020), AMVUTTRA (vutrisiran, 2022, with ATTR-CM label expansion in 2025), Leqvio (inclisiran, licensed to Novartis, 2020/2021), and Qfitlia (fitusiran, licensed to Sanofi, 2025). The company maintains a clinical pipeline of 25+ active programs targeting rare genetic diseases, cardiovascular, metabolic, hematology, and neuroscience indications, with a stated target of 40+ clinical programs under the Alnylam 2030 strategy.

Alnylam monetizes through three primary revenue streams: (1) direct commercial product sales of AMVUTTRA, ONPATTRO, GIVLAARI, and OXLUMO through its own specialty sales force and the Alnylam Assist patient services program; (2) licensing, milestone, and royalty revenue from out-licensed assets Leqvio (Novartis) and Qfitlia (Sanofi); and (3) strategic collaboration revenue from co-development deals with Roche (Zilebesiran), Regeneron (cemdisiran, ALN-APP), Vir Biotechnology (elebsiran), and others. In 2025 the company reported approximately $3.7 billion in total revenue with 81% gross margins and achieved its first year of GAAP profitability, while Q1 2026 net product revenue reached $1.036 billion (121% year-over-year growth) led by AMVUTTRA's $890 million quarterly sales following the ATTR-CM label expansion. The company employs more than 2,000 people, operates in 70+ countries with websites in 16+ languages, and is publicly traded on NASDAQ under the ticker ALNY since its 2004 IPO.

The principal commercial risk is concentration within the TTR franchise, where AMVUTTRA alone delivered approximately $910 million of the $1.036 billion in Q1 2026 product revenue, making diversification across the Leqvio (cardiovascular), Qfitlia (hematology), GIVLAARI (acute hepatic porphyria), and OXLUMO (primary hyperoxaluria) franchises a central strategic priority. Manufacturing is supported by a proprietary siRELIS enzymatic ligation platform and a new $250M Norton, Massachusetts facility announced in 2025, and research and development has been substantially augmented by a June 2026 partnership with Inceptive Nucleics applying generative AI to siRNA sequence and target design.

Name

Alnylam Pharmaceuticals

Legal name

Alnylam Pharmaceuticals, Inc.

Website

https://alnylam.com

Company type

Public

Founded year

2002

Operating status

Operating

Headcount range

1,001–5,000 employees

Short description

Alnylam Pharmaceuticals is a commercial-stage biopharmaceutical company that develops RNA interference (RNAi) therapeutics to silence disease-causing genes. It has six approved medicines across rare genetic, cardiovascular, and metabolic diseases and serves patients globally across 70+ countries.

Ownership category

akta.pro rank

Product category

Biopharmaceuticals / RNAi Therapeutics

NAICS

Pharmaceutical Preparation Manufacturing (325412), Biological Product (except Diagnostic) Manufacturing (325414), Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Pharmaceutical Preparations (2834), Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

siRNA Therapeutics (gene silencing) (HLAAADAB)

akta.pro secondary industries

RNA Delivery Technologies (LNPs, polymers, conjugates incl. GalNAc, peptides, exosomes) (HLAAADAD), RNA Chemistry & Oligonucleotide Engineering (backbones, modifications, stereochemistry) (HLAAADAE), RNA Therapeutics Platforms & Design (sequence design, target ID/validation, AI/bioinformatics) (HLAAADAG), RNA Manufacturing & CMC (oligo synthesis, IVT, purification, formulation, QC) (HLAAADAF), RNA Therapeutics Clinical Development (trial design, endpoints, patient stratification) (HLAAADAH)

Keywords

• RNAi therapeutics
• SiRNA medicines
• Rare disease drugs
• Biopharmaceutical platform
• RNA interference treatments

Headquarters

HQ city

Cambridge

HQ country

United States

HQ region

North America

Offices20 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Infrastructure

Revenue model

1. Commercial Product Sales: Direct sales of approved RNAi therapeutics: AMVUTTRA (vutrisiran), ONPATTRO (patisiran), GIVLAARI (givosiran), OXLUMO (lumasiran), and out-licensed Qfitlia (fitusiran, via Sanofi). Q1 2026 net product revenues reached $1.036B (121% YoY growth) with TTR franchise revenue of $910M (153% YoY).
2. Licensing & Royalty Revenue: Royalty and milestone revenue from out-licensed RNAi assets including Leqvio (inclisiran, licensed to Novartis for hypercholesterolemia — first approved RNAi therapeutic in a prevalent disease) and Qfitlia (fitusiran, licensed to Sanofi for hemophilia). Long-standing licensing model extending Alnylam's RNAi reach into prevalent diseases and other companies' commercial infrastructure.
3. Strategic Collaboration & Milestone Revenue: Upfront cash/equity, R&D funding, and potential milestone payments from strategic alliances such as the $2B Inceptive Nucleics AI discovery deal (June 2026, $30M upfront), the Tenaya Therapeutics $1.13B cardiovascular target discovery deal (March 2026, $10M upfront), and the Roche-partnered Zilebesiran Phase 3 program. Alnylam 2025 total revenue: ~$3B; TTM revenue: $4.29B (83% growth).

Pricing tiers

Go-to-market motion5 records

Distribution channels7 records

Marketing channels11 records

Core offering

Alnylam Pharmaceuticals discovers, develops, and commercializes RNA interference (RNAi) therapeutics — small interfering RNA (siRNA) medicines that selectively silence disease-causing genes by binding to messenger RNA (mRNA) and triggering catalytic degradation via the RNA-induced silencing complex (RISC). The company has six approved RNAi therapeutics (AMVUTTRA, ONPATTRO, GIVLAARI, OXLUMO, Leqvio via Novartis, Qfitlia via Sanofi) and a pipeline of 25+ clinical programs spanning transthyretin amyloidosis, cardiovascular, metabolic, hematology, and neuroscience indications.

Product overview

Alnylam Pharmaceuticals operates a platform-plus-portfolio architecture centered on its proprietary RNA interference (RNAi) therapeutic platform, supported by two validated siRNA delivery technologies (lipid nanoparticles/LNPs and GalNAc conjugates, plus a C16 conjugate platform for CNS delivery). The platform has yielded four wholly-owned approved RNAi medicines (ONPATTRO®, AMVUTTRA®, GIVLAARI®, OXLUMO®), two licensed approved medicines (Leqvio® licensed to Novartis for hypercholesterolemia, and Qfitlia™/fitusiran licensed to Sanofi for hemophilia), and a clinical pipeline of 25+ programs spanning transthyretin amyloidosis, cardiovascular, metabolic, hematology, and neuroscience indications. Patient-facing support programs (Alnylam Assist®, Alnylam Act®) and community programs (Alnylam Challengers) round out the offering, with manufacturing consolidated in Norton, MA (USA) and supported by the siRELIS™/Cyreli enzymatic ligation platform.

Differentiator

Problem solved

Functional benefit

Brands

• AMVUTTRA: Approved RNAi therapeutic (vutrisiran) for ATTR amyloidosis with cardiomyopathy and hATTR amyloidosis with polyneuropathy.
• ONPATTRO
• GIVLAARI
• OXLUMO
• Qfitlia
• Leqvio
• Alnylam Assist
• Alnylam Act
• Alnylam Challengers
• siRELIS

Products and services

• AMVUTTRA (vutrisiran) GalNAc-conjugated siRNA therapeutic administered subcutaneously quarterly for the treatment of ATTR amyloidosis with cardiomyopathy and hATTR amyloidosis with polyneuropathy. Supported by HELIOS-A and HELIOS-B Phase 3 data.
• ONPATTRO (patisiran) The world's first-ever approved RNAi therapeutic, an LNP-based siRNA therapeutic administered intravenously for hATTR amyloidosis with polyneuropathy, supported by the APOLLO Phase 3 study.
• GIVLAARI (givosiran) GalNAc-conjugated siRNA therapeutic administered subcutaneously for the treatment of Acute Hepatic Porphyria (AHP). Targets ALAS1 to reduce neurotoxic intermediate buildup.
• OXLUMO (lumasiran) GalNAc-conjugated siRNA therapeutic administered subcutaneously for the treatment of Primary Hyperoxaluria Type 1 (PH1). Supported by ILLUMINATE-A, -B, and -C Phase 3 studies.
• Leqvio (inclisiran) GalNAc-conjugated siRNA therapeutic for hypercholesterolemia, the first approved RNAi therapeutic in a prevalent disease. Licensed to Novartis for global commercialization. Supported by ORION-11 Phase 3 study.
• Qfitlia (fitusiran) Investigational RNAi therapeutic for hemophilia A or B. Licensed to Sanofi and approved in the U.S. in 2025. Supported by the ATLAS Phase 3 study.
• Alnylam Assist Comprehensive in-house patient support ecosystem staffed by Alnylam employees that supports patients receiving Alnylam therapies through insurance coverage understanding, financial support options, educational materials, and treatment access support.
• Alnylam Act Sponsored, no-charge, third-party genetic testing and counseling program for people in the U.S. and Canada with a family history or suspected diagnosis of hATTR amyloidosis, acute hepatic porphyria, or primary hyperoxaluria type 1. Has genotyped 125,000+ samples since launch.

Quantifiable outcome

• ~$3 billion 2025 total revenue (up ~81% YoY) and first GAAP profitability year
• +7 more outcomes

Named customers6 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Integration8 records

AI capability14 records

Feature7 records

Partnerships

19 partnerships are on record, tiered flagship, core and minor.

• Regeneron PharmaceuticalsflagshipOEM/ Whitelabel/ Licensing Partner · 22 June 2026Licensing agreement covering cemdisiran, an investigational siRNA therapeutic, for which Regeneron has accepted FDA Priority Review and EMA regulatory submissions for generalized myasthenia gravis (gMG) with target action date of November 2026. Regeneron is also developing cemdisiran in PNH and geographic atrophy. Additionally, Regeneron and Alnylam co-develop ALN-APP (mivelsiran) for Alzheimer's disease and cerebral amyloid angiopathy via Alnylam's C16 conjugate platform.
• GENESIS PharmacoreChannel Partner/ Reseller/ Distributor · 10 June 2026Strategic commercial partnership originally established in 2019 covering 13 Southeast European markets. Expanded in June 2026 to include 4 Nordic markets (Denmark, Finland, Norway, Sweden), bringing total coverage to 17 European markets. GENESIS Pharma commercializes Alnylam's RNAi therapeutics for serious cardiomyopathy conditions and rare genetic diseases across the expanded territory.
• Inceptive NucleicsflagshipStrategic or Co-development Partner · 3 June 2026Three-year strategic collaboration valued at up to $2 billion to accelerate RNAi therapeutics discovery. Combines Alnylam's RNAi platform and 20+ years of proprietary siRNA data with Inceptive's biology-trained AI foundation models to enhance siRNA design and predict top-performing therapeutic candidates. Includes $30 million upfront in cash and equity, with remaining payments tied to preclinical, regulatory, and commercial milestones. Alnylam's first AI-driven discovery partnership, supporting the Alnylam 2030 strategy.
• Viz.aicoreTechnology or Integration · 24 March 2026Strategic collaboration to develop an AI-enabled ATTR-CM care pathway that combines Viz.ai's FDA-cleared echocardiography AI algorithm (Us2.ai) with electronic health record integration and generative AI. The Viz Cardiac Amyloidosis Care Pathway will analyze echocardiograms, identify patients earlier, and guide clinicians through guideline-based diagnostic steps, with a multi-site pilot study at five pilot health systems.
• American Heart AssociationcoreGTM or Marketing Partner · 24 March 2026Alnylam is supporting a three-year American Heart Association initiative convening a 10-site multidisciplinary health system cohort to identify care gaps and scale effective models for diagnosing and managing ATTR-CM. The AWARE implementation study will begin at five pilot U.S. health systems later in 2026.
• Tenaya TherapeuticscoreStrategic or Co-development Partner · 5 March 2026Two-year research collaboration to discover and validate novel genetic targets for cardiovascular disease therapeutics. Tenaya will validate up to 15 gene targets using its target identification platform, and Alnylam will assume responsibility for all subsequent development and commercialization. Deal includes $10 million upfront plus potential milestone payments reaching up to $1.13 billion if all targets lead to approved therapeutics.
• HelixcoreTechnology or Integration · 5 March 2026Multi-year agreement granting Alnylam access to Helix's GenoSphere™ genomic and clinical datasets (including Exome+ sequencing data and longitudinal clinical records spanning an average of 13 years of EHR history across North American patients) to support Alnylam's drug discovery efforts in rare disease precision medicine.
• Komodo HealthcoreTechnology or Integration · 1 August 2025Strategic partnership to deploy the Marmot AI analytics platform across Alnylam's broader enterprise operations and customer-facing organization. Marmot maps over 330 million de-identified patient journeys and has reduced Alnylam's reporting cycles from months to hours while eliminating reliance on fragmented dashboards. The collaboration began in August 2025 and was expanded in June 2026.
• SanofiflagshipOEM/ Whitelabel/ Licensing Partner · 1 January 2025Qfitlia (fitusiran) for hemophilia A or B is licensed to Sanofi and was approved in the U.S. in 2025. Generates royalty and milestone revenue for Alnylam.
• RocheflagshipStrategic or Co-development Partner · 1 July 2023Strategic alliance with Roche AG to jointly develop and commercialize Zilebesiran, an investigational RNAi therapeutic for hypertension in patients with elevated cardiovascular risk. Currently in Phase 3 (KARDIA program); Alnylam-led development with US profit split and milestones/royalties ex-US. Phase 3 trial for zilebesiran was announced alongside Roche at ACC.26.
• NovartisflagshipOEM/ Whitelabel/ Licensing Partner · 1 January 2020Leqvio (inclisiran) for hypercholesterolemia is licensed to Novartis; the first approved RNAi therapeutic in a prevalent disease, approved in the EU in 2020 and U.S. in 2021. Generates ongoing royalty revenue for Alnylam.
• Our Future Health (OFH)coreStrategic or Co-development Partner · 1 January 2020Alnylam is a founding industry member of Our Future Health, the UK's largest ever health research programme aimed at transforming prevention, detection, and treatment of diseases.
• Regeneron Genetics CentercoreTechnology or Integration · 1 January 2020Partnership with Regeneron Genetics Center to leverage large-scale human genetic data for Alnylam's Human Genetics drug discovery platform, supporting genetically validated drug target identification.
• Vir BiotechnologycoreStrategic or Co-development Partner · 1 January 2019Strategic collaboration on elebsiran (an siRNA) for chronic hepatitis D / hepatitis delta virus (HDV) infection, where Vir is combining elebsiran with tobevibart. Alnylam received milestone payments from Arbutus (litigation) and has an ongoing elebsiran partnership with Vir via tobevibart combination.
• GENESIS Pharma (Southeast Europe)coreChannel Partner/ Reseller/ Distributor · 1 January 2019Original 2019 partnership covering 13 Southeast European markets for the commercialization of Alnylam's RNAi therapeutics for cardiomyopathy conditions and rare genetic diseases. Expanded to Nordic markets in 2026.
• UK Biobank Exome Sequencing Consortium (UKB-ESC)coreStrategic or Co-development Partner · 1 January 2017Alnylam is a founding member of the UK Biobank Exome Sequencing Consortium, leveraging large genetic datasets to validate genetically-informed drug targets through the Alnylam Human Genetics group.
• Massachusetts Biotechnology Council (MassBio)minorOthers · 1 January 2017Alnylam is a member of the Massachusetts Biotechnology Council (MassBio), benefiting from the New England life sciences cluster and academia. Alnylam was awarded MassBio's 2026 Scientific Impact Award.
• PreventionGeneticscoreImplementation/ SI/ Consulting Partner · 1 January 2014Third-party genetic testing partner that performs the Alnylam Act® sponsored, no-charge genetic testing services for hATTR amyloidosis, AHP, and PH1 in the U.S. and Canada. Patients and healthcare professionals interact directly with PreventionGenetics for test ordering and sample submission.
• Genome MedicalcoreImplementation/ SI/ Consulting Partner · 1 January 2014Third-party genetic counseling partner providing no-charge genetic counseling services to patients enrolled in the Alnylam Act program in the U.S. and Canada.

Scale indicators16 records

Recent moves8 records

Expansion highlights6 records

Direct peers

• Ionis Pharmaceuticals: Closest direct peer — Ionis is the pioneer of antisense oligonucleotide (ASO) therapeutics and the leading alternative gene-silencing platform to RNAi, with multiple approved products (Spinraza, Tegsedi, Waylivra) and a similarly structured rare-disease commercialization model. Competes head-to-head with Alnylam in TTR amyloidosis (with Tegsedi historically) and adjacent metabolic/neurology indications.
• Arrowhead Pharmaceuticals: Direct RNAi competitor with the TRiM platform and a pipeline spanning cardiometabolic, liver, and muscle targets; partnered with Takeda, Amgen, GSK, and others, and running multiple Phase 3 programs — represents the closest competing RNAi modality to Alnylam's GalNAc/LNP platforms.
• Wave Life Sciences: RNA therapeutics company developing stereopure oligonucleotide therapies, with a pipeline in Huntington's disease (WVE-003), DMD, and ALS; uses a different chemistry platform (PN chemistry) than Alnylam's siRNA but competes in similar CNS and rare-disease indications.

Broad incumbents

• Moderna: Larger, more diversified mRNA-based therapeutics company with commercial COVID-19 vaccine scale and a pipeline spanning oncology, rare diseases, and infectious disease; uses a related nucleic-acid modality (mRNA) rather than siRNA but represents a comparable platform-based biopharma with similar delivery-platform challenges.
• BioNTech: Major mRNA therapeutics company that, like Alnylam, built on foundational RNA biology and operates a platform-plus-pipeline model with oncology and infectious disease focus; comparable in scientific lineage (RNA biology platform) and European life sciences presence.
• Vertex Pharmaceuticals: Established commercial-stage rare-disease biopharma with multiple blockbuster franchises (Trikafta in CF) and a similar high-gross-margin/high-RD-intensity profile; Alnylam Director Stuart Arbuckle is former Vertex COO/CCO, and the companies compete for talent and capital in the rare-disease specialty biopharma segment.

Emerging players

• Regeneron Pharmaceuticals: Strategic partner (cemdisiran, ALN-APP) and a major biopharma developing antibody-based therapeutics with the Regeneron Genetics Center; while Regeneron is far larger and antibody-focused, it co-develops Alnylam's CNS assets and shares the rare-genetic-disease development thesis.
• Novartis: Strategic partner commercializing Leqvio (inclisiran) — the first RNAi therapeutic in a prevalent disease — globally; comparable as a major biopharma licensing RNAi platform assets but as a partner rather than a competitor for the broader cardiometabolic opportunity.
• BridgeBio Pharma: Genetics-driven rare-disease biopharma with a pipeline spanning TTR amyloidosis (acoramidis for ATTR-CM, directly competing with AMVUTTRA), and other Mendelian disorders; comparable as a competing modality (small molecule TTR stabilizer) in Alnylam's core ATTR-CM franchise.
• Intellia Therapeutics: In vivo CRISPR/Cas9 gene-editing company with clinical programs in transthyretin amyloidosis (NTLA-2001, a one-time TTR knockout) — directly threatening Alnylam's durable-silencing TTR franchise with a potentially curative one-time therapy that could displace chronic AMVUTTRA dosing.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat7 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xx.com/Alnylam
• YouTubeyoutube.com/@AlnylamPharmaceuticals
• Facebookfacebook.com/116396455088949
• LinkedInlinkedin.com/company/alnylam-pharmaceuticals
• Instagraminstagram.com/alnylampharma

Compliance5 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles16 records

Funding overview

Funding rounds9 records

Investors11 records

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M&A1 record

Investments

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Frequently asked questions about Alnylam Pharmaceuticals