Spotlight on a CSL Partner: Seattle Children's Research Institute

Back in 2020, we announced our partnership with this leading pediatric research center and shared our goal to develop stem-cell gene therapies for the life-threatening disease, Wiskott-Aldrich syndrome (WAS), along with plans to tackle other primary immunodeficiency diseases.

Researchers at Seattle Children’s are leading the way on WAS, a devastating genetic condition that affects young children. Female carriers exhibit no clinical signs, however males experience a myriad of problems, including difficulty with blood clotting, severe eczema and immunodeficiency, which can result in autoimmune disease, as well as greater susceptibility to cancer.

Without treatment, the one in 100,000 males born with WAS are unlikely to survive beyond the age of ten. Currently, the only known cure for WAS is a bone marrow transplant.

WAS is so named because of a mutation in the gene that produces the WAS protein in hematopoietic cells, critical for the development of a functional immune system. Seattle Children’s researchers have pioneered research into WAS. With CSL’s commitment to enhancing the quality of life for people with rare and serious diseases and aspirations in novel gene therapies, we hope to accelerate that research into clinical trials in the near future, with a therapy that could potentially correct the genetic abnormality.

Here’ what Dr. David Rawlings, Director of the Center for Immunity and Immunotherapies at Seattle Children’s Research Institute, said about our partnership: “Our alliance with CSL gives us access to world leading gene therapy technology, as well as the funding and technical expertise to translate our research into novel treatments that can offer hope for patients with rare genetic diseases.”

Now one of the world’s largest biotechs, a hallmark of CSL’s growth has been its approach to global collaboration, which is yielding new opportunities for innovative research with strategic alliances and partners around the world. As a global leader in biotechnology, our mission is to develop and deliver innovative medicines that save lives, protect public health and help people with life-threatening medical conditions to live full lives.

Across research hubs in Australia, the U.S. and Europe, CSL’s scientists are forging discoveries across our therapeutic areas of research focus: immunology, hematology, cardiovascular and metabolic, transplant, respiratory, and vaccines. Underpinning our work in these therapeutic areas of interest are our collective capabilities across four drug discovery platforms, including cell and gene therapy.

Over the past five years, our scientists and R&D experts have been developing a world-class cell and gene therapy platform. Collectively, CSL’s focus is on state-of-the-art technology and the development of ex vivo hematopoietic stem-cell gene therapy, which seeks to correct the underlying cause of disease, potentially leading to a cure.

Underpinning CSL’s rapid international expansion has been our substantive investment in research and development, to the tune of $4.1 billion globally over the past five years. We see long term, values-driven global alliances as vital to building our research and innovation capability. Our philosophy of global collaboration underpins our presence within research hubs and precincts around the world.

The CSL Research Acceleration Initiative establishes partnerships between CSL and global research organisations to progress the commercialization of promising discovery programs. Each year, the team led by Dr. Marthe D’Ombrain, Senior Director and Head of Global Research Innovation at CSL, seeks to identify promising research programs around the world, which will benefit most from fast-tracked collaboration and support.