ONE COMPANY with a vision to bring personalized gene therapy to the world.


Transforming patients' worlds

We strive to give people with lysosomal disorders freedom for life through gene therapies designed to halt or reverse disease with a single dose.

Gene therapy for the entire body

Our goal is simple and powerful: We aim to enable individuals with genetic disease to live longer — free from disease progression, free from painful and debilitating symptoms and free from the burden of chronic treatment regimens.

The life-limiting symptoms patients with these disorders experience are caused by the lack of a crucial functional protein. Our personalized approach to gene therapy starts with inserting a therapeutic gene in the patient’s own stem cells to enable production of the protein they lack. Over time, these cells are expected to multiply and produce daughter cells. Each new generation of daughter cells is expected to contain the therapeutic gene.

This approach holds the potential for the all-important protein to be durably expressed throughout the patient’s body, including the brain, delivering freedom for life.

Our initial focus is on lysosomal disorders, where the standard of care does not halt disease progression or adequately address many symptoms, particularly in the brain and nervous system. Our investigational therapies are currently being studied in clinical trials in Gaucher disease and Hunter syndrome. We are also advancing a program in Pompe disease.


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Our Pipeline

Learn more about our pipeline of investigational gene therapies.