New collaboration may allow researchers to develop potential gene therapies for pediatric diseases

A new collaboration between Seattle Children's Research Institute and bluebird bio, Inc., a biotechnology company based in Cambridge, Mass. and Seattle, will allow researchers to develop potentially transformative gene therapies for severe genetic and rare pediatric diseases. The collaboration supports pediatric researchers in Seattle who will work with bluebird bio scientists and the company's gene editing technology to research potential cures for genetic pediatric diseases.

Under the agreement, bluebird bio will provide a total of up to $3 million over three years to sponsor research performed at the Seattle Children's Research Institute Center for Immunity and Immunotherapies, which studies potential treatments for a variety of inherited blood diseases, including bubble boy disease, sickle cell disease and immune deficiency or marrow failure syndromes. The funding will be used to build gene editing tools to treat pediatric genetic defects using T-cell or hematopoietic stem cell-based immunotherapies and to support a five-person research team.

"If we can cure a genetic disease in an infant by targeting and editing the causative gene within the infant's own genome, we change the course of that person's life, preventing decades of healthcare burdens and costs," said Dr. David Rawlings, Director of the Center for Immunity and Immunotherapies at the Seattle Children's Research Institute. "This collaboration with bluebird bio's research scientists supports our gene editing work and will accelerate research toward clinical trials, putting this innovative technology to immediate use in promising pediatric research."

Genetic diseases are caused by mutations in genes. Seattle Children's researchers target those mutated genes and edit them to become normal. By targeting and repairing gene mutations, researchers seek to cure the diseases they cause. Researchers are also studying specific genes known to be involved in diseases and disrupting the genes to study them more precisely.

"The collaboration with the scientists at bluebird bio provides expertise and resources that can help us transition gene editing work from the lab to the clinical research setting," said Dr. Andrew Scharenberg, Co-director of the Center for Immunity and Immunotherapy Program in Cell and Gene Therapy in association with Rawlings and also a member of bluebird bio's Scientific Advisory Board. "The sooner we can catch and treat genetic diseases, the better the outcome will be for patients over time."

Seattle Children's is beginning a clinical trial this fall to study gene replacement therapy for Severe Combined Immune Deficiency, a genetic blood disease where patients lack normal immune function. In gene replacement—the gene therapy technology that is currently being studied in clinical trials in a variety of genetic disorders —researchers insert a working copy of a defective, disease-causing gene into a patient's cells.

Gene editing, in contrast, is the next and more advanced iteration of gene therapy. It aims to repair a patient's own gene instead of requiring insertion of a replacement gene. Because gene editing targets and repairs the genes in a person's own body, it may achieve a more natural level of function and be applied to a wider range of genetic diseases. These features of gene editing could potentially lead to safer long-term therapies.

Under the terms of the agreement, bluebird bio will provide Seattle Children's researchers with access to certain aspects of its gene therapy technology platforms, and bluebird bio will retain development rights over related future discoveries. In addition, bluebird bio has licensed intellectual property around several Seattle Children's Research Institute technologies.

Seattle Children's researchers will join bluebird bio and other organizations on Sept. 10 in Cambridge, Mass. at a forum organized by the Massachusetts Biotechnology Council. Seattle Children's Research Institute representatives will highlight their new model for joint funding of collaborative partnerships to promote translational research.