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Alnylam Pharmaceuticals, Inc.
As of May 21, 2026 at 10:37 UTC
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About Alnylam Pharmaceuticals, Inc.
Alnylam Pharmaceuticals is a leader in the study of RNA interference (RNAi) therapeutics. RNAi is a naturally occurring biological pathway within cells for sequence-specific silencing and regulation of gene expression. Alnylam's commercial RNAi therapeutic products include Onpattro and Amvuttra (for hATTR amyloidosis), Givlaari (for acute hepatic porphyria), and Oxlumo (for primary hyperoxaluria type 1), all developed and commercialized by Alnylam. Plus, Leqvio (for hypercholesterolemia) and Qfitlia (for hemophilia A or B), which are being commercialized by Alnylam's partners, Novartis and Sanofi, respectively. It also has several clinical programs across various therapeutic areas, including cardio-metabolic diseases, neuroscience, and hematology.
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Latest News
5 articlesThe global protein synthesis research market is experiencing accelerating growth driven by increased investment in cell and gene therapy research, rising chronic disease prevalence, and expanding healthcare budgets. North America leads with 42.8% market share, while Asia-Pacific infrastructure expansion presents significant growth opportunities. Key market drivers include strong late-stage therapy pipelines and government support through initiatives like the CHIPS and Science Act.
The oligonucleotides clinical trial landscape is expanding rapidly with 200+ companies developing 600+ pipeline drugs. Major players like Novartis, Alnylam Pharmaceuticals, Amgen, and WaVe Life Sciences are advancing promising therapies across various clinical stages. Approximately 20+ oligonucleotides are in late-stage development while 150+ are in mid and early stages. Recent developments include SanegeneBio's $110M Series B funding, FDA approvals for multiple orphan drug designations, and several companies advancing Phase II and Phase III trials.
Edgewise Therapeutics, a $3.1B biotech company, has two major drug candidates with significant market potential. EDG-7500 for hypertrophic cardiomyopathy uses a novel mechanism (promoting heart relaxation) with superior safety profile compared to existing treatments, with efficacy data expected by June 2026. Sevasemten targets Becker Muscular Dystrophy, a disease with zero approved treatments, with pivotal trial results expected Q4 2026. The stock has tripled from its 52-week low and faces two binary catalysts this year.
BridgeBio Pharma stock surged 12.60% following William Blair's initiation of coverage with an Outperform rating and $93.03 price target. The analyst highlighted the company's accelerating Attruby launch, near-term NDA submissions for disease-modifying therapies, and a strong pipeline in rare genetic diseases including achondroplasia and autosomal dominant hypocalcemia. BridgeBio's products are positioned as next-generation alternatives to Pfizer's market-leading tafamidis.
Tenaya Therapeutics shares surged 31.20% following a research collaboration agreement with Alnylam Pharmaceuticals focused on discovering genetic targets for cardiovascular disease treatments. Tenaya will validate up to 15 gene targets and receive up to $10 million in upfront payments with potential milestone payments reaching $1.13 billion. Despite the positive news, the stock remains in a bearish trend trading below its 100-day and 200-day moving averages.
