The brief
- 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
Silencing disease-causing genes
siRNAs bind to disease-causing mRNA and trigger their degradation, preventing protein production.
Reaching target tissues
GalNAc conjugates direct RNAi drugs to the liver via receptor-mediated endocytosis, allowing subcutaneous dosing.
Proven safety and efficacy
Four approved medicines show RNAi can deliver clinical benefit in rare genetic diseases.
Broadening to common diseases
RNAi programs advancing in diabetes, obesity, hypertension and neurological diseases.

