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.