Dedicated to making life better for people living with genetic diseases

Developing novel gene therapies by using lentiviral vector delivery platforms

Pursuing multiple indications in parallel by switching gene targets to create optimal therapies for a broad range of genetic diseases

"We are building a leading lentiviral-based cell and gene therapy company using our versatile dual technology platform and in-house manufacturing capabilities to advance development of potentially transformative therapies for patients and families seeking relief from devastating genetic diseases."

Matthew Gantz, President and Chief Executive Officer

Unlocking the potential of lentiviral vector delivery platforms

Using ex vivo and in vivo technologies to pursue multiple indications