Company Profile
Alnylam Pharmaceuticals
— An RNAi biotech that became profitable by delivering siRNA to the liver and now aims at ten tissue types
Alnylam PharmaceuticalssiRNA medicine using RNA (RNAi)Biotech First product ONPATTROLipid nanoparticles (LNP)siRNAJoin GalNAcI switched to a method to deliver to the liver by subcutaneous injection. Net income is US$3,394 million and net income is US$3,373 million.From the previous year's red to blackHome In this article, we will organize the generation of delivery technology and cooperation and manufacturing that support it.
- Alnailam and DDS in 30 seconds
- About logo and company name
- Where is this company in the world of drug discovery and DDS?
- Company size
- Drug discovery and DDS resources
- Past Initiatives
- Future plans
- What is DDS?
- Collaboration mapping of drug discovery and DDS
- Technologies, Products and Services
- The risk of this company
- Glossary/Source/Ins ment and Source Response Table
1. Standing position in the drug discovery and DDS
(siRNA)Biotech, a commercial stage for in-house sales
US$23,383 millionGalNAc binding siRNA. 1 subcutaneous injection in 3 months
Products of AlnailamHow to deliver to the liverdivided into two generations. First product ONPATTRO siRNADosage with droplets wrapped in lipid particles (LNP)AMVUTTRA・GIVLAARI・OXLUMO・LeqvioSubcutaneous injection by directly connecting the ligand with three GalNAcHome FactsHome In the sales of 2025, GalNAc type AMVUTTRA grows to US$23,384 million, while LNP type ONPATTRO decreased to US$17,279 million from the previous year to 32%. Facts。 The generation of the delivery method has been replaced as it isThis is the characteristic of this company.
2. Logo and company name
Alnailam Cube is a trademark of the company. In this article, we do not reproduce the generated image,
Images obtained from the official websiteHome img/alnylam_logo.svg Contact Us
Note: Corporate name Alnylam Pharmaceuticals, Inc.(Delaware Corporation) In Japanese, it is written as "Alnailam". The product name is written in the US name (AMVUTTRA, ONPATTRO, etc.). Leqvio (sold by Nov、s) and Qfitlia (sold by Sanofi) are drugs born from the technology of Alnailam.Sales partnersComment
Official Site:https://www.alnylam.com/3. Where is this company in the world of drug discovery and DDS?
TypeBiotech (siRNA)The company is described as a global commercial-stage biopharmaceutical company that develops new treatment drugs based on RNAi. Six first-in-class RNAi drugs have been approved. FactsHome For delivery, to the liverGalNAc binding or LNPFor the brain and spinal cordThe method combines decyl (C16) as a fat-soluble ligandIt explains that the delivery to the heart, skeletal muscle, and fat tissue is also progressing. FactsHome The mechanism of siRNA is the technical commentary "nucleic acid medicine", "LNP" is "lipid nanoparticles" and "blood brain barrier".
4. Company Scope
| Indicators (US Accounting Standards, US Dollars) | FY2024 | FY2025 | January to June 2026 |
|---|---|---|---|
| Sales | 2,248.2 | 3,713.9 | 2,458.1 |
| Net sales | 1,646.2 | 2,986.5 | 2,208.2 |
| Affiliated income | 510.2 | 553.4 | 129.2 |
| Royalty | 91.8 | 174.0 | 120.6 |
| R&D expenses | 1,126.2 | 1,319.8 | 778.0 |
| Net Income | △278.2 | 313.7 | 370.5 |
Our employeesApprox. 2,500(as of December 31, 2025), about 1,945 people in the United States and about 555 people outside the United States. About 270 people increased in 2025 FactsHome AMVUTTRA accounts for77.5% of product sales This paper comments。
AMBUTTRAATTR Cardio diseaseIn March 2025, U.S. sales increased to 1 year from the previous year. FactsHome In other words1 Dependence on product and 1 disease area (TTR) has increasedBlack
5. Drug discovery and DDS resources
| Resource Types | ation | |
|---|---|---|
| Person: Research Center | Cambridge headquarters and major research facilities (approx. 295,000 square feet) and additional research facilities (approx. 129,000 square feet). Rent | Facts |
| Person: | 2,500 people 2025 includes research, clinical, CMC (chemical, manufacturing and quality control). No selection | Facts |
| Gold: R&D expenses | US$1,978 million (2025). US$6,067 million Alnylam 2030’s policy to reinvest about 30% of sales (non-GAAP) research and development | Facts |
| Gold: Manufacturing Equipment | A GMP manufacturing facility in Norton, Massachusetts (approximately 200,000 square feet, operating under GMP in December 2020), and a GMP facility (approximately 15,000 square feet) to make ONPATTRO bulk preparation in Cambridge. In December 2025, the siRNA manufacturing base "siISIS" has been published. (The investment amount is not listed in the 10-K.) | Facts |
| External: Funding | Strategic capital alliance with Blackstone in April 2020. US$10 billion in consideration of Leqvio’s royalty, plus up to US$15,000 million in development of vutrisiran and zilebesiran | Facts |
| External: Technical Partnership | PeptiDream (July 2021, peptide-siRNA bonding deliveries outside the liver), Inceptive (Jun 2026, RNAi discovery using the generated AI model) | Facts |
| External: Acquisition | Acquisition of company in drug discovery and DDS field | Not specified |
| University/Research Organization/Public Fund | 10-K said, “I have a variety of affiliation and licenses with other companies and academic institutions to obtain delivery technology,” but the individual name and the amount of public funds are not included. | Not specified |
6. Past Initiatives
| Time | Home | The meaning of drug discovery and DDS |
|---|---|---|
| Year 2002 | As a dedicated RNAi drug | |
| Year 2012 | Established a manufacturing facility in Cambridge and established a GMP manufacturing system for patisiran (ONPATTRO) bulk preparation. | LNP preparation |
| February 2013 | The rights to RNAi drugs targeting PCSK9 were acquired by Sanofi in January 2020, along with the rights to Leqvio. | High cholesterol blood |
| 2014 year | Ex。 strategic alliance with Sanofi Revised in January 2018, Alnairum has made the worldwide rights of TTR products into the form that Sanofi has the right to fitusiran (Qfitlia) | Decision to make TTR in-house |
| Year 2015 | Ionis and oligonucleotides are mutually exclusive licenses for eight targets | Two large enterprises of nucleic acid drugs are patented |
| April 2019 | Global Partnership with Regeneron and RNAi Drugs with Eyes, Central Nerve and Some Liver Targets | Expanding outside the liver |
| April 2020 | Capital alliance with Blackstone (USD 10 billion + USD 10 million) | Cash Royalty Forward |
| December 2020 | Norton’s GMP manufacturing facility is running | In-house production of clinical original drugs |
| July 2021 | Partnership between PeptiDream and peptide-siRNA binding | Delivering peptides outside the liver |
| 2023 year | Co-development and co-sales agreement between Roche and zilebesiran. T contract fee US$3 billion | Challenges to disease with large number of patients |
| March 2025 | AMVUTTRA was approved in the US for ATTR Cardio disease (June) and TONC-M (September) of the next-generation TTR drug ucacresiran started.Home In the same month, Qfitlia sold by Sanofi is also approved by the United States. | GalNAc binding siRNA gains great indication |
| June 2025 | Started zilebesiran's Phase III ZENITH in September and received a $3 billion milestone from che. | IKARIA Technology and Cardio Outcome Test |
| December 2025 | Publication of Norton Expansion for Enzymatic Ligation Manufacturing "si IS" | To manufacture not only chemical |
| Early 2026 | Alnylam 2030 | Targeting over 40 clinical programs |
| April 2026 | Approx. 1,250 cases to 1,750 cases | Because incorporation is faster than expected |
| June and July 2026 | Collaboration with Inceptive and AI-generated drug discovery (June) Completion of incorporation of the phase II cAPPricorn-1 of mivelsiran (July) | Utilization of AI and verification of delivery to brain by C16 |
Clinical trial example: HELIOS-B (AMVUTTRA, ATTR myocardia)
March 2025 The number is quoted from the US attachment,Does not recommend specific treatment Facts。
| Test name | HELIOS-B(NCT04153149) |
| Adult of wild or hereditary ATTR myocardia. 40% use tafamijis when testing begins | |
| Dosage | AMVUTTRA 25mg subcutaneous injection (326 cases) vs placebo (328 cases) |
| Main evaluation items | A complex of all deaths and recurrence of cardio。 events (emergency checkup of cardio。 hospitals and cardio。 systems). Up to 36 months |
| Result | Decrease in risk compared to placebo is 28% in the entire population and 33% in groups that do not use taphamijis at the beginning of the trial |
7. Future plans
Annual rate 25% or moreNot a company's goal
Alnylam 2030 Undetermined / FutureHome The main items under development are:Not confirmed。
| Products | Target and Delivery Method | Stage | |
|---|---|---|---|
| nucresiran | ATTR Amyloidosis. IKARIA Technology (GalNAc binding) | TONC-M, TRITON-PN | Undetermined / Future |
| zilebesiran | High blood pressure (angiotensinogen target). ESC+ GalNAc binding | Developed (III phase ZENITH, cardio outcome test). Partner | Undetermined / Future |
| mivelsiran | Brain amyloid and Alzheimer's disease. C16 Binding to Brain | Phase II | Undetermined / Future |
| ALN-HTT02 | Ketinton disease. C16 binding. Partnership with Regeneron | Under development (phase Ib) | Undetermined / Future |
| cemdisiran | Systemic severity muscle asthenia. Regeneron | Application acceptance in the U.S. (approved by Regeneron in November 2026) | Undetermined / Future |
None of the following are listed in the primary information Undetermined / Future。
Norton extension (si IS)Investment and completion period/ 10 types of organizationsEnglish(Heart, skeletal muscle, fat, etc.)/ InceptiveProductsDate of approval(aiming to achieve the target time of ucacresiran). This article does not use media or analysts.
8. What is this company aiming for drug discovery and DDS?
This includes parts that the company does not directly state.The range of information that can be readand write with the basis.
| Read | Primary Information | |
|---|---|---|
| Established in the liver"Long-acting with subcutaneous injections" to other organizations | Alnylam 2030’s “10 Types of Organization” Targets and Delivery by C16, Peptides and Antibodies | |
| TTRyour own products with your next generation | The goal of ucacresiran to be released in 2028 by 2030 with a 30% royalty to Sanofi for AMVUTTRA sales | |
| Patients with a large number of diseasesMitigate risks with major stakeholders | Zilebesiran shares development costs with che 40 to 60% and Novo Nordisk for hypercholesterolemia, leading to Novo Nordisk's 2020. | Facts |
| Manufacturing costChange of the process itselfContact Us | siISIS (enzymatic ligation) is introduced to "promote production capacity and lower manufacturing costs" and 10-K is described. |
Alnailam is a contract of 2018, instead of obtaining the worldwide rights of TTR products from Sanofi,A step-by-step Royalty from 15% to 30% depending on AMVUTTRA’s global salesSanofi FactsHome 10-K also increased AMVUTTRA salesRising Royalty RateI wrote that the sales cost was increased from 18.6% of 2024 to 22.7% of 2025. FactsHome On the other hand, the next-generation productucucresiran uses its own IKARIA technology and in the phase I of the healthierReduced blood TTR by more than per year and maintained for 6 monthsand the company has announced FactsHome 、Technical improvements to reduce doses and switch to light products with loyalty burdenRead the two reasons why Undetermined / FutureHome However, ifucucresiran has a royalty to Sanofi, it is a guess.
9. Collaboration mapping of drug discovery and DDS
10. Technologies, Products and Services
| Product | Contact Us | 2025 sales (million USD) | Sales |
|---|---|---|---|
| AMVUTTRA（vutrisiran） | GalNAc binding (ESC). Subcutaneous injection per 3 months | 2,313.8 | Company |
| GIVLAARI（givosiran） | GalNAc binding | 308.5 | Company |
| OXLUMO（lumasiran） | GalNAc binding | 191.4 | Company |
| ONPATTRO（patisiran） | Lipid nanoparticles (LNP). Infusion | 172.8 | Company |
| Leqvio（inclisiran） | GalNAc binding | Royalty | Novartis |
| Qfitlia（fitusiran） | This article does not confirm the delivery method | Royalty | Sanofi |
Generation of delivery technology can be arranged in 10-K description as follows Facts。 ESCGalNAc binding of (stabilization chemistry) is expected to be administered once a month or twice a year, and the followingESC+the treatment area is spread by up to six times. MoreIKARIAis chemical modulation aiming for twice a year or once a year. Other than liverC16 (Hexadecyl) bindingPeptiDreamPeptide BondingContact Us
ONPATTRO attachment per mLIonized lipid DLin-MC3-DMA 13.0mg, DSPC 3.3mg, cholesterol 6.2mg, PEG lipids (PEG2000-C-DMG) 1.6mgHome 4 ingredients of LNP are written as it is FactsHome This is a typical four-。 formula used in this series of technical commentary "lipid nanoparticles". It was necessary to be given by drip. GalNAc binding AMVUTTRA formula9 slices, 6 sInclude FactsSulfur is a 2′-fluoro modifier, considered to be from phosphodiester binding Undetermined / FutureHome Adding to fatIt is difficult to decompose the itself by chemical modificationI understand that I moved to the direction. In terms of production, the supply of GalNAc ligand is a bottleneck, and 10-K has supplied the GalNAc material for partners, but the partners have moved to their production. FactsHome Further si、IS is a chemically synthesized short fragmentConnect with EnzymeWe explain how to synthesize long chains one by one, It is possible to reduce waste and refining load of raw materials Undetermined / Future。
11. Risks that this company has
| Risks | ||
|---|---|---|
| Focus on products | AM.5% of product sales in 2025 AMVUTTRA | This paper comments |
| Rising the Royalty burden | Increase the royalty rate to Sanofi (up to 30%) as AMVUTTRA sales increase. Increasing sales costs by 22.7%, and the company is expected to increase by 2026. | Facts |
| External dependence of original medicine | Agilent is the only supplier of AMVUTTRA, ONPATTRO, GIVLAARI original medicine. In the next few years, 10-K is listed if you rely on a contract manufacturer | Facts |
| TTR Market Competition | Ionis・AstraZeneca eplontersen cardio tests aiming for the same TTR were not achieved in July 2026 and 10-Q said. The market environment can change due to competitive trends | Facts |
| Regulatory and Legal Notices | In April 2026, FDA noted the general website of AMVUTTRA. Summon for government price report in October 2025. Prosecution of Cipla in patent infringement of Leqvio’s after-sale application (March 2026) | Facts |
| Unexpected delivery to outside the liver | C16・Peptide delivery items are in phase II or earlier and there is no approval example | Undetermined / Future |
12. Glossary
- RNA Interference
- A mechanism in which short two-strand RNA decomposes the mRNA of the array and suppresses the work of the gene.
- siRNA
- Short two-strand RNA for RNAi. All the drugs of Alnailam are in this form.
- GalNAc binding
- How to send siRNA to the liver by adding three sugars (N-acetyl galactosamine) that bind to the receptor of liver cells.
- LNP
- Attach a lipid-particle to the ionized nucleic acid with cholesterol, lipid, and PEG lipids. ONPATTRO
- C16 binding
- How to attach a decyl group (16 carbon chains) as a fat-soluble ligand and aim for delivery to the brain, spinal cord, and eyes.
- ESC／ESC+／IKARIA
- Generation name of siRNA chemical modification of alnilam. Aim to extend the dose interval to increase the specificity of the later generation.
- ATTR Amyloidosis
- A disease in which the protein called transthyretin (TTR) is attached to the organ. There is a neuro type and cardio type.
- Enzyme regeneration
- How to connect short nucleic acid fragments with s. si IS is used for manufacturing.
Concept diagram of AI generation13. Source
- Alnylam Pharmaceuticals Form 10-K for the) year ended December 31, 2025 Business overview, delivery technology (GalNAc, LNP, C16, ESC, ESC+, IKARIA), Alnylam 2030, terms of cooperation, manufacturing (Agilent, Norton, si IS), employee, facility, sales and cost breakdown. https://www.sec.gov/Archives/edgar/data/1178670/000162828026007497/alny-20251231.htm
- Alnylam Pharmaceuticals Form 10-Q for the quarterly period ended June 30, 2026 Sales, Cost, Net Income, Inceptive, sales revenue, TON-CM, Mivelsiran, cemdisiran, FDA https://www.sec.gov/Archives/edgar/data/1178670/000117867026000062/alny-20260630.htm
- Alnylam Pharmaceuticals "Alnylam Announces Partial Repurchase of 1.00% Convertible Senior Notes Due 2027" Established in 2002 and used to confirm the location of the head office. https://www.sec.gov/Archives/edgar/data/1178670/000119312525315047/d201569dex991.htm
- National Library of Medicine DailyMed AMVUTTRA(vutrisiran) Structure and si formula of GalNAc binding siRNA, design and result of HELIOS-B trial. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=8db0facb-81b6-4006-9239-27dc6409c5d3
- National Library of Medicine DailyMed ONPATTRO (patisiran) US attachment. 4 ingredients and content of lipid complexes. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=e87ec36f-b4b4-49d4-aea4-d4ffb09b0970
- National Library of Medicine DailyMed GIVLAARI (givosiran) US attachment. GalNAc binding siRNA and 2′-F・2′-OMe modifier. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=167e663c-11e1-497b-a3fc-951d65d58eaa
- Alnylam Pharmaceuticals Official website (for confirmation of company profile and logo) https://www.alnylam.com/
14. Response table of claims and sources
| Content | ||
|---|---|---|
| Official name, location of head office and international headquarters, Nasdaq listed, self-regulation of commercial stage biopharmaceu companies, approved 6 products | Facts | Source 1 https://www.sec.gov/Archives/edgar/data/1178670/000162828026007497/alny-20251231.htm |
| Established in 2002 | Facts | Source 3 https://www.sec.gov/Archives/edgar/data/1178670/000119312525315047/d201569dex991.htm |
| Sales and breakdown of 2024 and 2025, sales by product and region, R&D and breakdown, net profit and loss, sales cost rate | Facts | Source 1 https://www.sec.gov/Archives/edgar/data/1178670/000162828026007497/alny-20251231.htm |
| Sales, R&D, Net Income from January to June 2026 | Facts | Source 2 https://www.sec.gov/Archives/edgar/data/1178670/000117867026000062/alny-20260630.htm |
| About 2,500 employees, facilities (e.g. Cambridge Norton), manufacturing system (Agilent, Norton GMP operation, si IS extension) | Facts | Source 1 https://www.sec.gov/Archives/edgar/data/1178670/000162828026007497/alny-20251231.htm |
| Explanation of delivery technology (GalNAc, LNP, C16), ESC, ESC+, IKARIA, Phase I of ucresiran | Facts | Source 1 https://www.sec.gov/Archives/edgar/data/1178670/000162828026007497/alny-20251231.htm |
| Agreement with John Regeneron, Sanofi, Nov)s (MDCO), Blackstone, PeptiDream, Ionis, and Dicerna, the royalty rate to Sanofi | Facts | Source 1 https://www.sec.gov/Archives/edgar/data/1178670/000162828026007497/alny-20251231.htm |
| US approval in AMVUTTRA ATTR myocardia (March 2025), US approval in Qfitlia (March 2025) | Facts | Source 1 https://www.sec.gov/Archives/edgar/data/1178670/000162828026007497/alny-20251231.htm |
| Partnership with Inceptive (June 2026), expanded TON-CM cases, mivelsiran and cemdisiran progress, FDA calligraphy, subpoenas, prosecutions against Cipla, and references to the epilontersen trial | Facts | Source 2 https://www.sec.gov/Archives/edgar/data/1178670/000117867026000062/alny-20260630.htm |
| AMVUTTRA structure (GalNAc 3 residue, molecular formula sulfur9・6), HELIOS-B test design and results | Facts | Source 4 https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=8db0facb-81b6-4006-9239-27dc6409c5d3 |
| ONPATTRO's 4 fats and content | Facts | Source 5 https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=e87ec36f-b4b4-49d4-aea4-d4ffb09b0970 |
| GIVLAARI is GalNAc binding siRNA | Facts | Source 6 https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=167e663c-11e1-497b-a3fc-951d65d58eaa |
| Targets of Alnylam 2030 (reinvestment of ucresiran’s launch period, 10 organizations, growth of more than 25% annual rate, and reinvestment of approximately 30% of sales) | Undetermined / Future | Source 1. Not a company's goal https://www.sec.gov/Archives/edgar/data/1178670/000162828026007497/alny-20251231.htm |
| AMVUTTRA’s 5% of product sales | This paper comments | This document is calculated from sales by source 1 product |
| Classification of type 3 (pharmaceu) company / drug discovery biotech / contract development manufacturing / research) | This paper comments | This article does not mention. Company name is an example from the series of this company. |
| siISIS expansion investment amount and completion time, 10 organization breakdown, contract amount with Inceptive, and expected time of approval of development products | Undetermined / Future | Not listed in Sources 1 and 2. Not 'where' in this article |
| Reading that the form established in the liver is crossed into other tissues, and reading that the manufacturing cost is lowered by process change | Interpre of this article from the description of Source 1 | |
| Reasons to replace with your next generation product with TTR (Number of doses and royalty burden) | Interpre of this article from the terms of contract with Sanofi in Source 1 and description ofucucresiran | |
| How sulfur-type solvents and s come from 2′-F modifiers and phosphoate bonds, the benefits of -based regeneration | Explanation of this article from the description of source 4 and 6 and source 1 |
Last Updated:September 26, 2026/Troy Technical
The number of this paper is based on the statutory disclosure document (Form 10-K, 10-Q, 8-K) submitted by the United States Securities and Exchange Commission (SEC) and the US attachment (DailyMed). We do not use estimates and media-based numbers of survey companies. The "、ulation" section is an interpretation that the article reads from the primary information and is not an announcement of the company. This article is not intended for medical advice, but does not recommend the use of certain drugs.