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.

Leo, 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, and hepatic infectious diseases.

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.

Leo, 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, and hepatic infectious diseases.

LATEST NEWS

>700
Employees
8
Programs Currently in Clinical Development
>1k
Study Participants Dosed
>14k
Doses Administered
>20
Clinical Studies to Date
>3yrs
Longest Duration of Exposure

*Updated as of May 2017

*Updated as of May 2017

OUR FOCUS ON PATIENTS

Leo: 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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