Our Product Pipeline
Gene Therapy - True Personalized Medicine
Our objective is to develop and commercialize a next generation of products based on the transformative potential of gene therapy to treat patients with severe genetic and orphan diseases. Central to this effort is a collective determination within our company to provide these patients with hope for a better life in the face of limited or no long-term safe and effective treatment options.
Our approach represents an important new therapeutic modality for patients with severe genetic and orphan diseases with significant unmet medical need. We have two clinical-stage programs in development for childhood cerebral adrenoleukodystrophy (CCALD) and beta-thalassemia/sickle cell disease and a preclinical oncology program in the chimeric antigen receptor (CAR) T cells field under a collaboration with Celgene Corporation.
Our gene therapy process involves inserting genetic material into the patient's own cells ex vivo, then re-introducing the cells to the patient. For beta-thalassemia, sickle cell disease and adrenoleukodystrophy, we insert a functional copy of the malfunctioning gene into the patient's hematopoietic stem cells (HSCs), with the goal of genetically modifying a patient's own cells to fundamentally correct or address the genetic basis underlying a disease. In oncology, the gene therapy process targets a different cell type, called T cells. In this case, genetic sequences are inserted into a patient's own T cells and are intended to program the T cells to specifically recognize and attack cancer cells. In all cases, gene therapy has the potential to change the way patients are treated by correcting the underlying genetic defect that is the cause of their disease or by providing additional functionality that can eliminate the disease, rather than offering solutions that only address their symptoms. Accordingly, we believe gene therapy has the potential to provide transformative disease modifying effects with life-long clinical benefits based on a single therapeutic administration.