Patients & Families

what is gene therapy?

Our gene therapy process works by genetically modifying a patient’s own cells by adding a functional copy of the gene of interest. We have extensive expertise in viral vector design and manufacturing and transduction, which we have developed into a potent gene therapy platform with potentially broad applications in a wide variety of indications with significant medical needs.

We are committed to individuals and families affected by severe genetic and rare diseases including cerebral adrenoleukodystrophy (CALD), transfusion-dependent β-thalassemia (also known as β-thalassemia major) and severe sickle cell disease, conditions for which we have ongoing clinical studies.

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