Understanding RNAi

RNA interference (RNAi) is the core discovery that forms the therapies Alnylam is developing. It is recognized as a major scientific breakthrough—but how does it work, exactly?


Learn About How RNAi-Based Medicines Work


Learn about RNAi and our investigational therapies

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Alnylam is focused on 4 Strategic Therapeutic Areas (STArs): genetic medicines, cardio-metabolic diseases, infectious diseases, and central nervous system (CNS) and ocular diseases

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Alnylam is dedicated to providing support for affected individuals, families, and caregivers

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Interested in our clinical trials?

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Our Focus On You

A Family Living with
hATTR Amyloidosis
Get more information about hATTR Amyloidosis.
Ania: Living with
Get more information about porphyria.
A Family Living with
Primary Hyperoxaluria Type 1 (PH1)
Get more information about primary hyperoxaluria.

Access To Investigational Medicines

Alnylam is dedicated to developing new therapies that have a positive impact on patient health, and to serving patients, patient families and patient communities through education, empathy, and awareness. We understand that there are seriously ill patients who will not be eligible for our clinical trials and may not have options for alternative therapies, including investigational therapies in trials being conducted by other sponsors. In these circumstances, Alnylam will consider providing a requesting physician with pre-approval access to a specific Alnylam investigational medicine, for the treatment of an individual patient outside of a clinical trial, when certain conditions are met.

Alnylam Assist®

Alnylam Assist® offers you a number of personalized services throughout your treatment with an Alnylam product.

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Alnylam Act®

Through Alnylam Act®, healthcare professionals can request genetic testing and counseling for hATTR amyloidosis, acute hepatic porphyria, or primary hyperoxaluria type 1 for individuals meeting certain criteria.

Clinical Trials

Learn more about Alnylam-sponsored clinical trials.

Science Of RNAi

RNAi is a breakthrough in the understanding of how genes are regulated in cells.


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