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Biotechs Collaborate on More Precise Gene, Cell Cancer Therapies

Mountain bluebird

Mountain bluebird (A. Kotok)

23 August 2018. Two biotechnology enterprises developing cancer treatments are pooling their efforts to develop therapies aimed at more specific targets across a range of solid tumor types. The deal with bluebird bio — the company spells its name in all lower case — is expected to bring Gritstone Oncology $30 million immediately, as well as milestone payments later on.

The company bluebird bio in Cambridge, Massachusetts, develops gene and genome-editing therapies for a number of disease conditions including cancer and inherited disorders, such as sickle cell disease and beta-thalassemia. Most of the company’s therapies uses healthy genes delivered with benign viruses called lentiviruses.

The company’s cancer technology takes a patient’s own blood-producing stem cells from bone marrow and cultures them outside the body. The culturing process creates replacement genes for the mutated stem cells causing the disease. The replacement genes modify the chimeric antigen receptors or CARs on the individual’s T-cells, with these CAR T-cells then binding to targeted proteins and destroying cancer cells.

Gritstone Oncology in Emeryville, California creates cancer immunotherapies based on the specific DNA composition of a patients’ tumor called tumor-specific neo-antigens or TSNAs. The company’s technology first evaluates DNA from each patient’s tumor using genomic sequencing and bioinformatics to identify individual’s TSNAs. Gritstone then applies its own algorithms with artificial intelligence to identify the most likely TSNAs to activate an immune response, and delivers personalized synthetic TSNAs for the patient as a vaccine, either on their own or with other therapies. Among Gritstone’s own treatments are T-cell receptors, naturally occurring proteins modified to target other characteristic proteins on the surface of cancer cells.

In their agreement, Gritstone Oncology will provide bluebird bio with 10 specific targets across several types of tumors based on its artificial intelligence identification process, as well as T-cell receptors, or TCRs, in some cases aimed at the designated targets. For those targets, bluebird bio will develop cell therapies, and be responsible for subsequent clinical development, commercialization, and manufacturing. Gritstone’s target identification technology is expected to aid bluebird bio in defining appropriate participants in clinical trials of those treatments.

In return, bluebird bio is providing Gritstone with an immediate payment of $20 million and taking an equity stake in Gritstone, acquiring $10 million of its preferred shares. Gritstone will also be eligible for future unspecified development, regulatory, and commercial milestone payments, as well as royalties on sales of approved therapies.

“As we expand our immuno-oncology focus and portfolio,” says Philip Gregory, chief scientist at bluebird bio in a joint statement, “this collaboration with Gritstone Oncology is highly complementary to our goal of rapidly advancing novel cellular therapies with the potential to transform the lives of people with cancer, particularly in solid tumor indications.”

The deal is the second collaboration for bluebird bio on cancer treatments so far this month. As reported in Science & Enterprise, bluebird bio and Regeneron Pharmaceuticals agreed to develop new cancer treatments, with Regeneron taking a $100 million equity stake in bluebird bio.

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