Platform

Gene Therapy

Gene therapy has been an evolving field for the last 20 years that has been characterized by great hope and potential. Gene therapy represents a unique opportunity to change the way patients with severe genetic and orphan diseases are treated by addressing the underlying cause of their disease, rather than offering solutions that focus only on their symptoms. By correcting the underlying genetic defect, we believe gene therapy can provide transformative disease modifying effects—potentially with life-long clinical benefits based on a single therapeutic administration.

Clinical proof of concept has been reported in peer-reviewed and industry journals across numerous important diseases, including:

  • Beta-thalassemia
  • Adrenoleukodystrophy
  • Metachromatic Leukodystrophy
  • Wiscott Aldrich Syndrome
  • Severe Combined Immunodeficiency (SCID)
  • Retinal disease
  • Chronic lymphocytic leukemia
  • Acute lymphocytic leukemia
  • Hemophilia B
  • Human immunodeficiency virus (HIV)

A growing body of gene therapy-based clinical data, the establishment of regulatory guidelines to govern the development and approval of gene therapy products and increased investment from the biopharmaceutical industry suggest that the time is now for gene therapy to emerge as an important new therapeutic modality for patients with significant unmet medical need. Encouraged by these developments, we believe we are particularly well positioned to drive the continued advancement of gene therapy technology in treating severe genetic and orphan diseases.