A new class of gene therapy

Liver Diseases

Our liver disease programs include:

Liver disorders

Phenylketonuria (PKU)

Hemophilia A

Wilson disease

Gaucher disease

Antibody gene therapy

Retinal Diseases

Our retinal disease programs includes:

Eye disorders

Leber’s congenital amaurosis 10 (LCA10)

Stargardt disease

Wet age-related macular degeneration (wet AMD)

Skeletal Muscle

Muscle disorders

Discovery

The CNS

Brain disorders

Discovery

Oncology

Cancer

Discovery

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