Re-dosable gene therapy

Liver disorders

We are focused on creating a diverse portfolio of programs for diseases of the liver. We have demonstrated preclinical proof of concept of systemic delivery targeted to the liver and are advancing toward the clinic with multiple therapeutic candidates formulated in lipid nanoparticles.

Liver disorders
Secreted Proteins

Hemophilia A

Inborn Errors of Metabolism

PKU

Areas of interest

We are creating delivery systems for additional tissues, including the eye, muscle and brain.

EYE DISORDERS

Liver disorders

LCA10

Stargardt

MUSCLE

Liver disorders

Exploratory

BRAIN

Liver disorders

Exploratory

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