Pipeline
Learn more about our investigational RNAi therapeutics in various stages of clinical development and their disease targets.
Any content about investigational therapeutics or investigational uses of FDA-approved products does not establish the safety or efficacy of these therapeutics or uses, and there is no guarantee of FDA approval.
Alnylam pipeline therapeutics that are out-licensed are not represented on this page. For further information, please see Alnylam's Clinical Development Pipeline.
For more information on any of our pipeline assets or clinical trials, please connect with an MSL or visit our clinical trials site.
Phase 3 Studies
Phase 2 Studies
Mivelsiran (ALN-APP)*
Cerebral Amyloid Angiopathy
Phase 1 Studies
ALN-BCAT*
Hepatocellular Carcinoma
ALN-HTT02*1
Huntington's Disease
ALN-TTRsc04*
ATTR Amyloidosis
Mivelsiran (ALN-APP)*
Alzheimer's Disease
