Science

Therapeutic Focus Areas

  • Alpha-1 Antitrypsin Deficiency

    Committed to Alpha-1 Patients

    Alpha-1 antitrypsin deficiency (AATD) is the most common hereditary disorder in adults. It is associated with an increased risk of developing pulmonary emphysema and liver disease, and also skin problems (panniculitis) and vasculitis.

  • Graft Versus Host Disease

    Fighting Against GVHD

    The allogeneic transplantation of hematopoietic cells (HCT) is a well-established treatment for hematologic diseases that cannot be cured with conventional treatments.The most common life-threatening complication is Graft Versus Host Disease (GVHD).

  • Lung Transplantation

    Advancing a novel clinical program to prevent lung rejection

    A lung transplant is a surgical procedure to replace a diseased or failing one or both lungs with a healthy lung, usually from a deceased donor. A lung transplant is…

  • Rabies

    Helping to prevent one of the deadliest diseases in the world

    Rabies is a preventable infectious viral disease that is almost always fatal following the onset of clinical symptoms. In up to 99% of cases, domestic dogs are responsible for rabies…

  • Type 1 Diabetes

    Exploring new potential therapy in new-onset type 1 diabetes

    Type 1 diabetes (T1D) is an autoimmune disease in which insulin-producing beta cells in the pancreas are mistakenly destroyed by the body’s immune system. T1D seems to have a genetic…