Our Focus

integrated product platforms
with broad therapeutic potential

We are leading the gene therapy revolution. Our integrated product platforms encompass gene therapy, cancer immunotherapy and gene editing – providing us with the potential to treat, and hopefully cure, a broad range of serious diseases.

integrated product platforms

Our objective is to develop and bring to market the most advanced products based on the transformative potential of gene therapy as a one-time treatment option for patients with severe genetic and rare diseases and cancer. At the core of this effort is our collective determination to provide these patients hope for a better life in the face of limited or no long-term safe and effective treatment options.

program pipeline