Alnylam develops RNA interference medicines that silence disease‑causing genes using chemically modified siRNAs delivered via proprietary platforms such as GalNAc conjugates for liver targeting and lipid nanoparticles for systemic use. Its portfolio includes FDA‑approved treatments for rare genetic and protein‑misfolding disorders and a pipeline of additional indications, with revenue from product sales, technology licensing, and patient‑support services.
Funding
$661.3M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Many diseases are driven by the overproduction of harmful proteins due to the expression of specific genes, and existing therapies often cannot directly target the underlying genetic cause, leaving patients with limited treatment options. This is especially acute for rare genetic disorders where conventional drug development is challenging.
Solution
Alnylam leverages RNA interference (RNAi) to develop therapeutics that selectively silence disease‑causing genes at the mRNA level, preventing the synthesis of pathogenic proteins. The company designs chemically optimized small interfering RNAs (siRNAs) and pairs them with proprietary delivery platforms—such as GalNAc conjugates for liver targeting and lipid nanoparticle formulations for systemic distribution—to achieve efficient, tissue‑specific gene knockdown. Approved products (e.g., patisiran, givosiran, lumasiran, inclisiran, vutrisiran, fitusiran) demonstrate the clinical viability of this approach, while a robust pipeline addresses both rare and more prevalent indications. Alnylam also supports patients through dedicated assistance programs, genetic‑testing services, and investigator‑initiated studies, ensuring broader access and ongoing scientific validation.
Target Audience
Primary customers are physicians, specialists, and healthcare systems treating patients with genetic or protein‑misfolding disorders, as well as the patients themselves who require targeted RNAi therapies.
Features
- siRNA design platform employing chemically modified nucleotides for enhanced stability and potency
- Targeted delivery technologies: GalNAc conjugates for hepatocyte uptake and lipid nanoparticle carriers for extra‑hepatic delivery
- FDA‑approved RNAi medicines covering transthyretin amyloidosis, acute hepatic porphyria, primary hyperoxaluria, and hypercholesterolemia
- Extensive intellectual‑property portfolio covering siRNA chemistry, delivery modalities, and specific gene targets across major markets (US, EU, Japan)
- Integrated patient‑support ecosystem (Alnylam Assist®, Alnylam Act®) offering insurance navigation, financial assistance, and free genetic testing
- Platform for investigator‑initiated studies and collaborative research to expand clinical evidence and explore new indications
- Scalable GMP manufacturing facilities enabling rapid production of clinical‑grade RNAi therapeutics