The alliance will initially focus on the development of treatments for patients with either Wiskott-Aldrich syndrome or X-linked agammaglobulinemia, rare life-threatening diseases. CSL and Seattle Children's will apply novel gene therapy technology to accelerate research towards clinical trials. Together, the organizations will investigate applying proprietary platform technology, Select+ and Cytegrity, to several preclinical gene therapy programs. Broadly, the research will be associated with commercialization of stem cell therapies, including consistent manufacturing, efficacy, and tolerability.
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