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Posted On Sep 6 2018 by

pharmaceuticals companies

Pharmaceuticals companies

To those who say “impossible, impractical, unrealistic,” we say:

CHALLENGE ACCEPTED

We’re relentless in our pursuit of new treatments because patients shouldn’t have to wait for hope.

Pharmaceuticals companies

We are developing a new class of innovative medicines called

RNAi THERAPEUTICS

that we believe have the potential to transform the lives of people living with rare diseases.

Pharmaceuticals companies

We are focused on diseases for which treatment options are limited or simply don’t exist.

OUR ROBUST PIPELINE

includes investigational RNAi therapeutics for rare genetic, cardio-metabolic, and hepatic infectious diseases.

Pharmaceuticals companies

To those who say “impossible, impractical, unrealistic,” we say:

CHALLENGE ACCEPTED

We’re relentless in our pursuit of new treatments because patients shouldn’t have to wait for hope.

Pharmaceuticals companies

We are developing a new class of innovative medicines called

RNAi THERAPEUTICS

that we believe have the potential to transform the lives of people living with rare diseases.

Pharmaceuticals companies

We are focused on diseases for which treatment options are limited or simply don’t exist.

OUR ROBUST PIPELINE

includes investigational RNAi therapeutics for rare genetic, cardio-metabolic, and hepatic infectious diseases.

LATEST NEWS

*Updated as of May 2017

Pharmaceuticals companies

Pharmaceuticals companies

Pharmaceuticals companies

Pharmaceuticals companies

Pharmaceuticals companies

Pharmaceuticals companies

*Updated as of May 2017

Pharmaceuticals companies

Pharmaceuticals companies

OUR FOCUS ON PATIENTS

Pharmaceuticals companies

Leo: Living with hATTR Amyloidosis

Pharmaceuticals companies

Rose Colin: Living with Porphyria

Pharmaceuticals companies

Venkat: Living with Hemophilia

PIPELINE

Pharmaceuticals companies

Late Stage

Learn more about our investigational therapies in hereditary ATTR amyloidosis, hemophilia, and acute hepatic porphyrias.

Pharmaceuticals companies

Early Stage

Learn more about our investigational therapies across multiple disease areas.

WORK WITH US

Pharmaceuticals companies

WORK WITH US

Join Us

We are looking for innovators to join our team—help us realize our commitment to developing a new class of medicines for patients with limited or inadequate options.

Pharmaceuticals companies


Last Updated on: September 6th, 2018 at 4:30 am, by


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