Alnylam Pharmaceuticals, Inc. (ALNY) is a global, leading biopharmaceutical company that specializes in discovering, developing, and commercializing RNA interference (RNAi) therapeutics.
Founded in 2002 in Cambridge, Massachusetts, the company was established with a bold vision to harness the potential of RNAi therapeutics. The inspiration for Alnylam's name came from the brightest star in the constellation, Lyra, symbolizing their commitment to scientifically innovative approaches and novel therapies.
RNAi is a natural cellular process of gene silencing, exploiting the body's own natural processes to prevent the production of specific proteins that contribute to various diseases. Alnylam's advancement and dedication in harnessing this cutting-edge science have put them at the forefront of the RNAi therapeutics industry.
Since its inception, Alnylam has remained committed to a patient-focused mission. This is evident in their diverse and robust pipeline of investigational medicines in both clinical and pre-clinical stages targeting severe and life-threatening diseases. These include rare genetic, cardio-metabolic, hepatic infectious, central nervous system (CNS), and ocular diseases.
Alnylam's pioneering work has led to the approval and marketing of ONPATTRO (patisiran), the world's first approved treatment for hATTR amyloidosis in adults. This achievement puts the company on the map as one of those that translated the Nobel Prize-winning science into an approved treatment, ushering in a new class of innovative medicines.
In their quest for scientific innovation, Alnylam also maintains strong collaborations with leading global pharmaceutical and life science companies, such as Sanofi Genzyme, Regeneron, and The Medicines Company. Through these strategic partnerships, the company aims to broaden the global reach of its RNAi therapeutics.
Alnylam Pharmaceuticals, Inc., although a publicly traded company (NASDAQ: ALNY), remains dedicated to its initial vision for scientific innovation, patient-focused drug development, and building an industry-leading pipeline of transformative RNAi-based medicines. Its more than 800-strong global workforce is driven by an unwavering commitment to patients in urgent need of treatments, reflecting the company's overarching dedication to the advancement of human health.
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City Therapeutics, an RNAi biotech co-founded by Alnylam founder Maraganore, has filed for an initial public offering (IPO) on Nasdaq under the ticker CTY. The company, which has raised approximately $238.8 million since its launch in October 2024, currently employs 81 people and has established partnerships with Bausch + Lomb and Biogen. The IPO aims to build on the success of Alnylam Pharmaceuticals Inc.

Alnylam Pharmaceuticals has updated its 2026 TTR franchise revenue outlook to $4.2 billion–$4.5 billion, reflecting a shift in market mix towards 80% first-line and 20% second-line use for AMVUTTRA. Despite the revision, the company emphasizes strong market fundamentals with many undiagnosed patients and upcoming pipeline catalysts in Huntington's disease, obesity, and bleeding disorders. Alnylam also noted that AMVUTTRA's deep TTR knockdown and quarterly dosing contribute to its market momentum.

Oppenheimer has reiterated its Outperform rating on Alnylam Pharmaceuticals (ALNY) with a $350 price target, following the release of new data that reinforces the potential of Nucresiran and the TRITON-CM study. This positive outlook comes despite recent adjustments to Alnylam's price target by Oppenheimer and RBC, and a cut in its 2026 net product revenue guidance. The company, which specializes in RNA interference technology, reported mixed Q2 2026 results but continues to advance its pipeline in treating rare and chronic diseases.

Alnylam Pharmaceuticals (ALNY) has seen a significant 46.8% fall in its stock price over the past year, making it appear cheap. However, its valuation, particularly its P/E ratio of 40.8x, suggests it is still expensive compared to industry averages and its calculated fair P/E of 32.4x. Investors are looking to upcoming clinical data at the ESC Congress 2026 for AMVUTTRA and zilebesiran to see if these can justify the premium valuation despite recent weaker returns.
Alnylam Pharmaceuticals (ALNY) has seen a significant stock price decline of 46.8% over the past year, yet its current valuation still appears expensive based on earnings. The company's P/E ratio of 40.8x is higher than the biotech industry average, suggesting investors are paying a premium despite upcoming clinical data potentially impacting sentiment. The article questions whether future earnings can justify this premium or if the market will adjust expectations.