GLOBAL
Media Inquiries
Christine Lindenboom
SVP, Investor Relations & Corporate Communications
media@alnylam.com
About Alnylam
Founded in 2002, Alnylam was built upon a bold vision of turning scientific possibility into reality and is now advancing a new class of innovative medicines to address the needs of patients who have limited or inadequate treatment options. Our pipeline is focused on 4 therapeutic areas: genetic diseases, cardio-metabolic diseases, hepatic infectious diseases, and central nervous system (CNS) & ocular diseases.
About Alnylam
Founded in 2002, Alnylam was built upon a bold vision of turning scientific possibility into reality and is now advancing a new class of innovative medicines to address the needs of patients who have limited or inadequate treatment options. Our pipeline is focused on 4 therapeutic areas: genetic diseases, cardio-metabolic diseases, hepatic infectious diseases, and central nervous system (CNS) & ocular diseases.
Resources
Corporate Backgrounder
A synopsis of our RNAi therapeutics, experience, and active pipeline.
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Corporate Fact Sheet
A brief overview of our mission, products, and pipeline.
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Corporate Presentation
Additional details on our programs, products, and disease focus areas.
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Patient Access Philosophy
Our Patient Access Philosophy is our commitment to making the therapies we develop available to those who will benefit from them.
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Patient Access Philosophy Report
The Patient Access Philosophy Report is our annual read-out on our commitment to strive toward broad patient access to our medicines.
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Alnylam Assist®
An overview of our patient support program for U.S. residents.
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The Science of RNAi
RNA interference (RNAi) is a breakthrough in understanding how genes are regulated in cells. It also represents a completely new approach to drug discovery and development.
RNAi Therapeutics, A New Class of Medicine
How RNAi-Based Medicines Work
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RNAi Fact Sheet
Learn about the history of RNAi and what it means for the advancement of human therapeutics.
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Disease Information
About Acute Hepatic Porphyria
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Acute hepatic porphyria (AHP) refers to a family of rare, genetic diseases characterized by potentially life-threatening attacks and, for some patients, chronic debilitating symptoms that negatively impact daily functioning and quality of life. AHP comprises 4 subtypes, each resulting from a genetic defect leading to deficiency in one of the enzymes of the heme biosynthesis pathway in the liver: acute intermittent porphyria, hereditary coproporphyria, variegate porphyria, and ALAD-deficiency porphyria.
About Hereditary ATTR Amyloidosis
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Hereditary transthyretin-mediated (hATTR) amyloidosis is a rapidly progressive and life-threatening disease that is caused by a mutation in the transthyretin (TTR) gene. TTR protein is produced primarily in the liver and is normally a carrier of vitamin A. The mutation results in the accumulation of amyloid deposits in multiple organs of the body, including the nerves, heart, and gastrointestinal tract. The condition can have a debilitating impact on a patient’s life and may lead to premature death within 4.7 years following diagnosis.
About Primary Hyperoxaluria Type 1
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Primary hyperoxaluria (PH) constitutes a group of rare inherited disorders of the liver characterized by the overproduction of oxalate, an end-product of metabolism. There are 3 types of PH: type 1 (PH1), type 2 (PH2), and type 3 (PH3). PH1 is the most common and severe form, accounting for 70% to 80% of all cases. PH1 affects approximately 4 individuals per million in the United States and Europe, with an estimated 1,300 to 2,100 diagnosed cases. In some regions, such as the Middle East and North Africa, the genetic prevalence of PH1 is higher.
Products and Pipeline
Our pipeline of investigational RNAi therapeutics is focused on diseases with high unmet medical need that fall under 4 Strategic Therapeutic Areas (STArs): genetic diseases, cardio-metabolic diseases, hepatic infectious diseases, and central nervous system (CNS) & ocular diseases. We have multiple programs in late-stage and early-stage clinical development.
Lumasiran
Our investigational therapeutic for the treatment of primary hyperoxaluria type 1 (PH1).
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Vutrisiran
Our investigational therapeutic for the treatment of ATTR amyloidosis.
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Clinical Trials
Our clinical studies allow us to evaluate whether potential new treatments are safe and effective.
APOLLO-A and -B Phase 3 Studies
APOLLO-A evaluated the efficacy and safety of patisiran in patients with hereditary transthyretin amyloidosis (hATTR amyloidosis) with polyneuropathy, and APOLLO-B is evaluating patisiran in patients with transthyretin amyloidosis with cardiomyopathy (ATTR amyloidosis).
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ENVISION Phase 3 Study
The ENVISION Phase 3 study evaluated the efficacy and safety of givosiran in patients with acute hepatic porphyria (AHP).
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ILLUMINATE-A, -B, and -C Phase 3 Studies
ILLUMINATE-A randomized, double-blind, placebo-controlled Phase 3 study evaluating safety and efficacy of lumasiran in children (age 6 or older) and adults with PH1. ILLUMINATE-B open-label, multicenter Phase 3 study evaluating safety and efficacy of lumasiran in infants and young children (under the age of 6) with PH1. ILLUMINATE-C open-label, multicenter Phase 3 study evaluating safety and efficacy of lumasiran in PH1 patients of all ages with advanced renal disease, including patients on dialysis.
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HELIOS-A and -B Phase 3 Studies
Evaluating the efficacy and safety of vutrisiran in patients with hereditary transthyretin amyloidosis (hATTR amyloidosis) and with transthyretin amyloidosis cardiomyopathy (ATTR amyloidosis with cardiomyopathy).
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Click here to see all of our clinical studies.
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