CHALLENGE ACCEPTED
We're relentless in our pursuit of new treatments because patients shouldn't have to wait for hope.
RNAi THERAPEUTICS
that we believe have the potential to transform the lives of people living with rare diseases.
OUR ROBUST PIPELINE
includes investigational RNAi therapeutics for rare genetic, cardio-metabolic, and hepatic infectious diseases.
>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.
WORK WITH US
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.
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