Re-dosable gene therapy
We are committed to enabling generations of people with genetic diseases to live long, full lives.
To do this, we are harnessing our unique construct and delivery technologies to move beyond the limitations of current approaches and create therapies for liver disorders along with a wide range of other diseases.
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.
Areas of interest
We are creating delivery systems for additional tissues, including the eye, muscle and brain.