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Biotech Licenses Gene Editing for Autoimmune Disorders

Crispr-Cas9 illustration

Crispr-Cas9 illustration (

27 September 2017. A company developing therapies with gene editing technologies is acquiring a new form of gene editing on T-cells in the immune system to treat autoimmune diseases. The deal licensing gene editing techniques to biotechnology enterprise Casebia Therapeutics could bring Seattle Children’s Research Institute $12 million or more.

The agreement covers processes that employ the technology known as Crispr — an acronym for clustered regularly interspaced short palindromic repeats — a genome-editing process based on bacterial defense mechanisms that use RNA to identify and monitor precise locations in DNA. The actual editing of genomes by Crispr harnesses enzymes that cleave DNA strands at the desired points, with Crispr-associated protein 9, or Cas9, being the enzyme used most often.

The processes in this case are developed by researchers at Seattle Children’s that use Crispr to edit genes in regulatory T-cells in the immune system, which in autoimmune disorders fail to control effector T-cells that attack healthy cells and tissues in the body, instead of invading pathogens from outside. Examples of autoimmune disorders are type 1 diabetes, rheumatoid arthritis, lupus, psoriasis, Crohn’s disease, and multiple sclerosis.

The deal will give Casebia Therapeutics in Cambridge, Massachusetts an exclusive worldwide license to discoveries of immunologists David Rawlings and Andrew Scharenberg at Seattle Children’s. Rawlings is director of the Center for Immunity and Immunotherapies at Seattle Children’s, and Scharenberg, who recently joined Casebia as the company’s chief scientist, was co-director of the hospital’s cell and gene therapy program.

Casebia Therapeutics and Seattle Children’s will collaborate on development of Crispr-based treatments for a range of autoimmune diseases, with the research funded by Casebia. “This collaboration,” says Rawlings in a joint statement, “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.”

Over the course of the agreement, Casebia is expected to pay $12 million or more to Seattle Children’s. No further financial details were disclosed.

Casebia Therapeutics is already developing a Crispr-based therapy for severe combined immunodeficiency, or “bubble-boy” disease, an autoimmune condition where children are born in effect without a working immune system and require total isolation to prevent infections. The company was formed in December 2015 as a joint venture between drug maker Bayer and and biotechnology enterprise Crispr Therapeutics, with each company owning 50 percent of Casebia.

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