CSL Behring and Seattle Children's partner to develop gene therapies for primary immunodeficiency diseases

Reviewed by Emily Henderson, B.Sc.Jun 2 2020

Seattle Children's Research Institute, one of the top pediatric research institutions in the world, and global biotechnology leader CSL Behring announced a strategic alliance to develop stem cell gene therapies for primary immunodeficiency diseases.

Initially, the alliance will focus on the development of treatment options for patients with two rare, life-threatening primary immunodeficiency diseases - Wiskott-Aldrich Syndrome and X-linked Agammaglobulinemia. These are two of more than 400 identified primary immunodeficiency diseases in which a part of the body's immune system is missing or functions improperly.

CSL Behring will collaborate with Seattle Children's experts to apply our novel gene therapy technology to their research pipeline, with an aim to address unmet needs for people living with certain rare primary immunodeficiency diseases. Expanding our gene therapy portfolio into an area of immunology well known to CSL exemplifies how we are strategically growing our capabilities in this strategic scientific platform and are collaborating with world class institutions to access innovation with the potential to vastly improve patients' lives."

Bill Mezzanotte, MD, Executive Vice President, Head of Research and Development for CSL Behring

"Stem cell gene therapies that correct the genetic abnormality driving a child's disease will transform the therapeutic options for children with Wiskott-Aldrich Syndrome, X-Linked Agammaglobulinemia and other immunodeficiency diseases," said David J. Rawlings, MD, director of the Center for Immunity and Immunotherapies and division chief of immunology at Seattle Children's, and a professor of pediatrics and immunology at the University of Washington School of Medicine. "The collaboration with CSL Behring supports our longstanding research programs for pediatric immunodeficiency diseases and will accelerate this research toward clinical trials, helping get these innovations to the children who need them."

CSL Behring researchers, working with researchers from Seattle Children's Research Institute, will investigate applying the proprietary platform technologies, Select+™ and Cytegrity™, to several pre-clinical gene therapy programs. These technologies, which have broad applications in ex vivo stem cell gene therapy, are designed to address some of the major challenges associated with the commercialization of stem cell therapy, including the ability to manufacture consistent, high-quality products, and to improve engraftment, efficacy and tolerability.

Wiskott-Aldrich Syndrome (WAS) has an estimated incidence between one and 10 cases per million males worldwide, according to the National Institutes of Health. WAS patients' immune systems function abnormally, making them susceptible to infections. They also experience eczema, autoimmunity and difficulty forming blood clots, leaving them vulnerable to life threatening bleeding complications. Today the only known cure for WAS is a stem cell transplant, if a suitable donor can be found.

X-linked Agammaglobulinemia (XLA) is another rare primary immunodeficiency in which patients have low levels of immunoglobulins (also known as antibodies), which are key proteins made by the immune system to help fight infections. Like WAS, XLA affects males almost exclusively, although females can be genetic carriers of the condition. While no cure exists for XLA, the goal of treatment is to boost the immune system by replacing missing antibodies and preventing or aggressively treating infections that occur, according to the Immune Deficiency Foundation.