Casebia Therapeutics Announces License And Collaboration Agreements With Seattle Children's Research Institute

Exclusive license agreement and research collaboration aims to advance gene-edited regulatory T-cell therapies

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Casebia Therapeutics, a leading genetic engineering company, and Seattle Children’s Research Institute today announced an exclusive license agreement and research collaboration to explore new methods to treat and prevent autoimmune disease using CRISPR/Cas9 gene-edited regulatory T cells (Tregs)—a type of white blood cell that controls and modulates the body’s immune response.

The goal of the Casebia-Seattle Children’s research collaboration is to advance novel therapeutic strategies to develop gene-edited Tregs across a range of autoimmune conditions. Under the terms of the license and collaboration agreements, Casebia will sponsor and collaborate with Seattle Children’s in the research and application of gene-edited Tregs, and will receive exclusive worldwide rights to develop and commercialize specific intellectual property related to the collaboration. Over the course of the collaboration agreement, funding contributions to Seattle Children’s could exceed $12 million.

This effort builds on pioneering discoveries by David Rawlings, M.D., Director of the Center for Immunity and Immunotherapies at Seattle Children’s, and Andrew Scharenberg, M.D., who recently joined Casebia as Chief Scientific Officer from Seattle Children’s. Rawlings, Scharenberg and others at Seattle Children’s Program in Cell and Gene Therapy have made important strides in developing new technologies for engineering, manufacturing and expanding T cells for therapeutic applications.

This collaboration between Seattle Children’s and Casebia will accelerate the work that David Rawlings and I have led over the last decade to create new ways of selectively engaging the immune system in the fight against a wide range of diseases,” said Scharenberg, who previously served as the co-director of the Program in Cell and Gene Therapy and as principal investigator in the Center for Immunity and Immunotherapies at Seattle Children’s. “Tregs represent a potentially significant improvement to current therapies for autoimmune disorders, which induce broad-scale immune system suppression that limits their therapeutic utility. I am thrilled to combine this promising work with the gene editing know-how and resources at Casebia.”

Rawlings, who also serves as Chief of the Division of Immunology and Director of the Program for Cell and Gene Therapy at Seattle Children’s, and is a professor of pediatrics at the University of Washington School of Medicine, added: “The opportunity with Casebia represents a major step forward in Seattle Children’s goal to develop a new generation of cell therapies to treat children suffering from autoimmune diseases. This collaboration will allow us to much more rapidly leverage a series of new technologies and knowledge that we have developed for precisely editing and expanding a patient’s own regulatory T cells. Use of these cells in adoptive therapy is designed to restore immune tolerance and eliminate disease.”

Casebia was founded as a joint venture between Bayer and CRISPR Therapeutics to harness the potential of CRISPR/Cas9 gene-editing technology and develop new medicines for people with genetic and inherited diseases. The company is concentrating its efforts on making an impact in the areas of ophthalmology, non-malignant hematology, autoimmune disease and cardio-vascular disorders. This collaboration with Seattle Children’s will further develop Casebia’s autoimmune disease program, increasing the chances to offer new treatments to patients. Together, Casebia and Seattle Children’s will explore the full scope of engineered Tregs to treat serious diseases.

We believe that Seattle Children’s deep experience in the cell and gene therapy space, coupled with our own work in gene editing, will further enable our bold aspiration to develop treatments and cures for genetic diseases, and positively impact patients’ and their families’ lives,” said Jim Burns, Ph.D., President and Chief Executive Officer of Casebia.

At Seattle Children’s, we believe that one of the best ways to rapidly translate scientific innovation into treatments is by partnering with companies committed to the same goals. We hope to apply our joint scientific advances to provide long-term benefit and potential cures in our patients,” said Rawlings.

About Casebia Therapeutics
Casebia Therapeutics is a novel joint venture between Bayer and CRISPR Therapeutics, focused on discovering, developing and commercializing CRISPR/Cas9 gene-editing therapeutics to treat the genetic causes of bleeding disorders, autoimmune disease, blindness, hearing loss and heart disease. Formed in March 2016, Casebia has access to gene-editing technology from CRISPR Therapeutics in specific disease areas, as well as access to protein engineering expertise and relevant disease know-how through Bayer. Casebia is a free-standing entity, equally owned by Bayer and CRISPR Therapeutics, with its own scientific leadership and management team. The company’s Board of Directors has equal composition from Bayer and CRISPR Therapeutics. Casebia’s primary base of research operations is in Cambridge, MA, with a second site in San Francisco, CA.

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