Pipeline & Clinical Trials
Discover our pipeline of RNAi-based therapies. Learn more about these therapeutics, including clinical trial information.
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.
For more information on any of our pipeline assets or clinical trials, please connect with a Medical Science Liaison (MSL) or visit our clinical trials site.
Transthyretin Amyloidosis (TTR)
Overview
Nucresiran is an investigational drug under clinical development for ATTR amyloidosis with cardiomyopathy. Safety and efficacy have not been established.
Overview
Nucresiran is an investigational drug under clinical development for hATTR amyloidosis with polyneuropathy. Safety and efficacy have not been established.
Cardiovascular (CV)
Overview
Zilebesiran is an investigational drug under clinical development for hypertension. Safety and efficacy have not been established.
Program management:
Partnered, Alnylam-led development with US profit split and milestones/royalties ex-US
Overview
Zilebesiran REVERSIR is an investigational drug under clinical development for hypertension. Safety and efficacy have not been established.
Program management:
Partnered, Alnylam-led development with US profit split and milestones/royalties ex-US
Metabolic
Overview
ALN-4324 is an investigational drug under clinical development for type 2 diabetes mellitus. Safety and efficacy have not been established.
Overview
ALN-2232 is an investigational drug under clinical development for obesity and weight management. Safety and efficacy have not been established.
Neuroscience
Cemdisiran is an investigational therapeutic being developed for the treatment of myasthenia gravis by Regeneron Pharmaceuticals, Inc. For any questions about cemdisiran, please contact Medical.information_Global@regeneron.com
Overview
Mivelsiran is an investigational drug under clinical development for cerebral amyloid angiopathy (CAA). Safety and efficacy have not been established.
Overview
Mivelsiran is an investigational drug under clinical development for Alzheimer's Disease. Safety and efficacy have not been established.
Overview
ALN-HTT02 is an investigational drug under clinical development for Huntington's disease. Safety and efficacy have not been established.
Program management:
Partnered, Alnylam-led with profit split
Overview
ALN-5288 is an investigational drug under clinical development for Alzheimer’s disease. Safety and efficacy have not been established.
Program management:
Partnered, Alnylam-led with profit split
ALN-SOD is an investigational therapeutic being developed for the treatment of SOD1 amyotrophic lateral sclerosis (ALS) in partnership with Regeneron Pharmaceuticals, Inc. For any questions about ALN-SOD, please contact Medical.information_Global@regeneron.com
ALN-SNCA is an investigational therapeutic being developed for the treatment of Parkinson's disease by Regeneron Pharmaceuticals, Inc. For any questions about ALN-SNCA, please contact Medical.information_Global@regeneron.com
Hematology
Cemdisiran is an investigational therapeutic being developed for the treatment paroxysmal nocturnal hemoglobinuria by Regeneron Pharmaceuticals, Inc. For any questions about cemdisiran, please contact Medical.information_Global@regeneron.com
Overview
ALN-6400 is an investigational drug under clinical development for hereditary hemorrhagic telangiectasia. Safety and efficacy have not been established.
Overview
ALN-6400 is an investigational drug under clinical development for Von Willebrand Disease. Safety and efficacy have not been established.
AG-236 is an investigational therapeutic being developed for the treatment of polycythemia vera by Agios Pharmaceuticals. For any questions about AG-236, please contact medinfo@agios.com
ALN-CFB is an investigational therapeutic being developed for the treatment of paroxysmal nocturnal hemoglobinuria by Regeneron Pharmaceuticals, Inc. For any questions about ALN-CFB, please contact Medical.information_Global@regeneron.com
Other
Cemdisiran is an investigational therapeutic being developed for the treatment of geographic atrophy by Regeneron Pharmaceuticals, Inc. For any questions about cemdisiran, please contact Medical.information_Global@regeneron.com
Elebsiran is an investigational therapeutic being developed for the treatment of hepatitis D by Vir Biotechnology, Inc. For any questions about elebsiran, please contact info@vir.bio
Overview
ALN-BCAT is an investigational drug under clinical development for hepatocellular carcinoma. Safety and efficacy have not been established.
Overview
ALN-4285 is an investigational drug under clinical development. Safety and efficacy have not been established.
Overview
ALN-4915 is an investigational drug under clinical development. Safety and efficacy have not been established.
ALN-F1202 is an investigational therapeutic being developed by Regeneron Pharmaceuticals, Inc. For any questions about ALN-F1202, please contact Medical.information_Global@regeneron.com
MED-US-Pipeline-2600008
