AstraZeneca, Sernova join forces on cell therapy research

Several diseases will be targeted in preclinical research project

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Note: This story was updated May 8, 2023, to remove hemophilia as one of the specific indications the collaboration will focus on, as these have not yet been publicly disclosed.

AstraZeneca and Sernova are teaming up for a preclinical research project that aims to develop new cell therapies for several diseases.

“Sernova has developed a novel cell therapy approach for the potential treatment of insulin-dependent diabetes, hypothyroidism and hemophilia A,” Philip Toleikis, Sernova’s president and CEO, said in a press release.

Hemophilia is caused by genetic mutations that interfere with the production of blood-clotting proteins — factor VIII in the case of hemophilia A, and factor IX in the case of hemophilia B.

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There are many other disorders that are caused by abnormally low levels of specific proteins. For example, type 1 diabetes is an autoimmune disease characterized by a chronic deficiency in insulin, a hormone that’s important for regulating blood sugar levels.

One potential treatment strategy for these types of disorders is cell therapy. The general idea is that cells could be engineered in the lab to produce high levels of the missing protein, and then transplanted into a patient, where they can produce the protein continually to restore its levels.

The transplant step is a substantial hurdle in the development of cell therapies, however, because newly transplanted cells face many unique challenges. For example, newly transplanted cells initially lack access to blood vessels that deliver them nutrients, and they may be recognized as abnormal and destroyed by the host’s immune cells.

Sernova is developing a technology called the Cell Pouch System that aims to overcome these obstacles. The system consists of an implantable medical device, made with materials that have been used in other approved implantable devices, that is designed to provide a safe and supportive environment for transplanted cells to grow.

“By engrafting functional therapeutic cells within an implanted Cell Pouch, which naturally [connects with the blood vessels of] surrounding tissues, we create an environment for the production and release of absent or under-expressed hormones and proteins,” Toleikis said.

Under the new agreement, AstraZeneca will be leading preclinical work to explore the use of this system for some of its experimental cell therapy research platforms. The companies will decide on next steps based on the results of these experiments.

“We are pleased to be working with AstraZeneca in the preclinical assessment of its various proprietary therapeutic cells in combination with our Cell Pouch,” Toleikis said.