Developing Therapies To Transform Lives

Developing Therapies To Transform Lives

Astellas Gene Therapies is an Astellas Center of Excellence developing genetic medicines with the potential to deliver transformative value for patients.

Watch the story of a family affected by a neuromuscular disease

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.

X‐linked Myotubular Myopathy
AT132
Gene replacement therapy candidate to treat XLMTM, a disease caused by mutations in the MTM1 gene, which encodes the protein myotubularin
Pompe Disease
AT845
Gene replacement therapy candidate to treat Pompe disease caused by mutations in the gene encoding the lysosomal enzyme GAA
Myotonic Dystrophy Type 1
AT466
AAV-antisense candidate to treat DM1 caused by a mutation in the DMPK gene, resulting in the accumulation of toxic DMPK RNA in affected cells.

 

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

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STATE-OF-THE-ART FACILITIES

The science of genetic medicines is complex. Our robust capabilities and industry leading manufacturing facilities put us at the forefront of developing innovative therapies.

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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.

MEET OUR TEAM

JOIN OUR TEAM

 

OUR PEOPLE

 

 

COMPANY UPDATES

 

@AstellasGeneTx

Learn more about what we’re doing to become the leader in the development and commercialization of innovative genetic medicines, the people who make us who we are, and the exciting job opportunities available by following us on LinkedIn.

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Learn more about what we’re doing to become the leader in the development and commercialization of innovative genetic medicines to treat serious rare neuromuscular diseases, the people who make it great and the exciting job opportunities available by following us on LinkedIn.

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