We are partnering with private companies to rapidly develop treatments, devices and technologies that improve children’s lives. Our latest partnerships include:

Collaborating to Advance Immunotherapy Research Efforts

Seattle Children’s Research Institute is partnering with Nexgenia, a privately held biotechnology company focused on the development and manufacture of proprietary polymer-based reagents, to develop improved immunotherapies for the treatment of cancer. The collaboration will allow researchers from both institutions to use Nexgenia’s technology platform to efficiently target and select immune cells, including CAR T cells. Funding for the partnership came from the Seattle Children’s Research Institute Office of Science–Industry Partnerships.

Under terms of the agreement, Nexgenia researchers will prepare cell selection reagents using the company’s polymers and magnetic nanoparticles, combined with antibodies provided by Dr. Michael Jensen of Seattle Children’s Research Institute. Both labs plan to test the combined reagents at small scale, using cells lines. Selected reagents will then be tested at full preclinical scale. Following this work, Nexgenia reagents may be advanced for clinical evaluation. Learn more.

Pioneering Genome Editing and Gene Therapy Research

Dr. David Rawlings and Dr. Andrew ScharenbergDrs. David Rawlings and Andrew Scharenberg

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 diseases. The collaboration supports pediatric researchers in Seattle who will work with bluebird 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 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.” Learn more.

Innovative Therapies for Autoimmune Diseases

Dr. Anne Stevens Dr. Anne Stevens

Seattle Children’s Research Institute and Kineta Inc., a Seattle-based biotechnology company, joined forces in 2014 to study a potential therapy for autoimmune diseases.

Kineta developed a drug candidate, ShK-186, that is in Phase 1B trials for adult autoimmune disorders. Seattle Children’s is funding the research institute’s Dr. Anne Stevens, a pediatric rheumatologist, to work with Kineta scientists on in vitro studies of the drug’s effect on kidney cells from pediatric patients with lupus nephritis. If those studies are successful, the team plans to launch phase 1 studies of ShK-186 in children.

The studies could ultimately illuminate ShK-186’s side effects and what dose to use in children – and potentially lead to the first pediatric lupus therapy ever approved by the Food and Drug Administration. This gives Kineta the opportunity to help children with autoimmune diseases, and developing a pediatric application of ShK-186 means Kineta can extend its patent on the adult drug, potentially increasing profits.

The research institute and Kineta have also launched pre-clinical studies of ShK-186 for pediatric multiple sclerosis, inflammatory bowel disease and asthma.

Destroying Cancer Cells with Immunotherapy

The research institute is a key partner in Juno Therapeutics Inc., a biotechnology firm that has raised more than $300 million to develop novel cell-based cancer immunotherapies. These therapies use chimeric antigen receptor and high-affinity T-cell receptor technologies to genetically engineer T cells to recognize and kill cancer.

These treatments reduce longer-term toxicities associated with current chemotherapeutics and provide potential cures even for patients with widespread disease. The research institute is leading clinical trials for pediatric patients with relapsed acute lymphoblastic leukemia and neuroblastoma.