INNOVATIVE THERAPIES

Our investigational therapies target rare diseases through three modalities: gene replacement, exon skipping gene therapy and vectorized RNA knockdown. We are leveraging Astellas’ global resources, industry leadership in immune biology, and deep scientific expertise to expand our reach and develop valuable new genetic medicines
for patients around the world.

COURAGEOUS PATIENTS

We depend on courageous patients and their families to advance our investigational therapies. Our patient communities are important partners and collaborators, informing our clinical trial programs and drug development process.

COURAGEOUS PATIENTS

OUR PEOPLE

We take pride in our mission to develop transformative genetic medicines to patients around the world. Our experienced team has the passion and drive to accomplish our goal of helping patients with life-threatening diseases. If you’re looking for a fast-paced work environment where employees whole heartedly embody a “Get Stuff Done” attitude, you’ll fit right in.

 

OUR PEOPLE

 

 

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