Alnylam Pharmaceuticals

RNA interference, RNAi medicines, Amvuttra, TTR amyloidosis

USpublic~2,500 staffest. 2002alnylam.com (opens alnylam.com)Checked 10 Oct

The brief

Analyst view
  • Alnylam commercialized RNAi technology, filing four approved medicines by 2023.
  • Amvuttra hit $1.01B quarterly revenue in Q2 2026, up 106%, about 15 months after ATTR-CM launch.
  • Pipeline includes next-gen nucresiran, mivelsiran (Alzheimer's), and programs for diabetes, obesity.

Technical approach

As reported
RNAi mechanism

Silencing disease-causing genes

siRNAs bind to disease-causing mRNA and trigger their degradation, preventing protein production.

Drug delivery

Reaching target tissues

GalNAc conjugates direct RNAi drugs to the liver via receptor-mediated endocytosis, allowing subcutaneous dosing.

Clinical validation

Proven safety and efficacy

Four approved medicines show RNAi can deliver clinical benefit in rare genetic diseases.

Pipeline expansion

Broadening to common diseases

RNAi programs advancing in diabetes, obesity, hypertension and neurological diseases.

Primer