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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.

Learn More About Alnylam ›

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Resources

Corporate Backgrounder

A synopsis of our RNAi therapeutics, experience, and active pipeline.

(Format: PDF)

Corporate Fact Sheet

A brief overview of our mission, products, and pipeline.

(Format: PDF)

Corporate Responsibility Report

Our Corporate Responsibility Report details our approach to corporate responsibility and current and planned efforts in our five key areas of focus.

(Format: PDF) (Format: PDF) (Format: PDF)

Patient Access Philosophy

Our annual readout on progress against the pillars of our Patient Access Philosophy.

(Format: PDF) (Format: PDF) (Format: PDF) (Format: PDF)

Alnylam Assist®

An overview of our patient support program for U.S. residents.

(Format: PDF)

Corporate Logos

Our company logo in various sizes.

(Format: Zip 523 KB)

Alnylam Rare Disease Trend Report

Our Rare Disease Trend Reports are based on survey responses from >30 U.S. payer and plan decision-makers and provides a unique window into the minds of insurers about orphan drug access.

(Format: PDF) (Format: PDF) (Format: PDF)

RNAi Fact Sheet

Learn about the history of RNAi and what it means for the advancement of human therapeutics.

(Format: PDF)

RNAi Activity Booklet for Kids

Learn about the basic science of RNAi through fun activities such word search, connect the dots, matching, and coloring. This activity booklet is intended for families and children aged 7-14.

(Format: PDF)

Management Board

Yvonne Greenstreet, MBChB

Chief Executive Officer

Timothy Maines

Chief Technical Operations and Quality Officer

Tolga Tanguler

Chief Commercial Officer

Pushkal Garg, MD

Chief Medical Officer and EVP, Development & Medical Affairs

Jeff Poulton

Chief Financial Officer

Kelley Boucher

Chief Human Resource Officer

Piyush Sharma, JD

Chief Ethics & Compliance Officer

Kevin Fitzgerald, PhD

Chief Scientific Officer

Evan Lippman

Chief Corporate Development & Strategy Officer

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.

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RNAi Therapeutics, A New Class of Medicine

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RNAi Therapeutics, A New Class of Medicine

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RNAi Therapeutic Process

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Disease Information

About Acute Hepatic Porphyria

Download Fact Sheet265.66 KB (Format: PDF)

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 ATTR Amyloidosis

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Transthyretin-mediated (ATTR) amyloidosis is an underdiagnosed, rapidly progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins. The misfolded TTR proteins collect as amyloid deposits throughout the body, including the nerves, heart and digestive system, resulting in progressive organ damage. There are two different types of ATTR amyloidosis – hereditary ATTR (hATTR) amyloidosis, which is caused by an inherited variant, or change, in the TTR gene, and wild-type ATTR (wtATTR) amyloidosis, which occurs without a TTR gene variant.

About Primary Hyperoxaluria Type 1

Download Fact Sheet332.29 KB (Format: PDF)

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.

About Hypertension

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Hypertension, also known as high blood pressure, is a complex multifactorial disease clinically defined by most major guidelines as a systolic blood pressure (SBP) of above 140 mm Hg or a diastolic blood pressure (DBP) greater than 90 mm Hg, though some guidelines have a lower threshold of a SBP above 130 mm Hg or a DBP greater than 80 mm Hg. More than one billion people worldwide live with hypertension, and fewer than 20 percent of those affected have it under control.

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.

ONPATTRO® (patisiran)

Now available in the United States.

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GIVLAARI® (givosiran)

Now available in the United States.

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OXLUMO® (lumasiran)

Now available in the United States.

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AMVUTTRA® (vutrisiran)

Now available in the United States.

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Zilebesiran

Investigational therapeutic for the treatment of hypertension.

 

ALN-APP

Investigational therapeutic for the treatment of Alzheimer’s Disease

 

See the Rest of Our Pipeline ›

Clinical Trials

Our clinical studies allow us to evaluate whether potential new treatments are safe and effective.

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APOLLO and APOLLO-B Phase 3 Studies

APOLLO evaluated the efficacy and safety of patisiran in patients with the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis, and APOLLO-B is evaluating patisiran in patients with the cardiomyopathy of wild-type transthyretin-mediated (wtATTR) amyloidosis or hATTR 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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KARDIA Phase 2 Studies

Evaluating the efficacy and safety of zilebesiran as monotherapy across different doses administered quarterly and biannually in patients with mild-to-moderate hypertension, and when used in combination with conventional antihypertensive medications.

Click here to see all of our clinical studies.

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