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R&D Transplant


Despite the remarkable advances in solid organ transplantation reflected by improved patient and graft survival rates, the treatment and management of solid organ transplant recipients remains a complex area of modern medicine. Organ transplant recipients are required to take life-long immunosuppressive medication to prevent their own immune systems from rejecting the donor organ (allograft). While current standard of care is very effective in the prevention and treatment of acute rejection, chronic rejection remains a high unmet medical need and is responsible for most cases of allograft failure. Our plasma derived C1 Esterase Inhibitor (C1-INH) product is currently being investigated in a clinical trial to treat one form of rejection (antibody mediated rejection) in kidney transplant recipients.

picture of a gowned doctor in operating theatre operating on a person

Haematopoietic stem cell transplantation (HCT) is a potentially curative treatment option for patients with malignant or non-malignant haematologic diseases. Acute graft-versus-host disease (GVHD) is a serious condition that arises as a complication in recipients of HCT from allogeneic donors. GVHD is a leading cause of mortality and morbidity in allogeneic HCT. Currently, few therapeutic options exist for the prevention of GVHD and all patients undergoing HCT typically receive immunosuppressive medications. These agents also increase the risk of infection and/or contribute to increases in relapse mortality due to enhanced immunosuppression. As such, there is a significant medical need to develop effective, less toxic preventative therapies for acute GVHD. We are currently investigating the potential of our plasma derived product Alpha-1 Antitrypsin (AAT) for the prevention of acute GVHD in patients receiving an allogenic HCT.

We are driven by our promise to bring lifesaving therapies to patients with rare disease and we are partnering with leading experts and worldwide regulatory authorities to deliver these novel immunomodulatory therapeutics to the field of transplantation.