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.

Rose, living with Porphyria

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.

Dane, living with hATTR amyloidosis

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, hepatic infectious diseases, and CNS diseases.

 

NOW APPROVED IN THE EU

 

NOW APPROVED IN THE U.S.

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.

Rose, living with Porphyria

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.

Dane, living with hATTR amyloidosis

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, hepatic infectious diseases, and CNS diseases.

LATEST NEWS

>800
Employees
7
Programs Currently in Clinical Development
1
Approved Product*
>20k
Doses Administered
>25
Clinical Studies to Date
>4yrs
Longest Duration of Exposure

Updated as of August 2018
* In the U.S. and European Union

Updated as of August 2018
* In the U.S. and European Union

OUR FOCUS ON PATIENTS

A Family Living with hATTR Amyloidosis

Rose & Colin: Living with Porphyria

Venkat: Living with Hemophilia

OUR

PIPELINE

Late Stage

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

Early Stage

 

Learn more about our investigational therapies across multiple disease areas.

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.

View Job Openings >

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