The βAir is a platform technology that could be adopted to sustain various cell-based therapies for long periods of time. Similar with the T1D application, the βAir could incorporate engineered or primary cells of either allogeneic or xenogeneic origin that would produce essential bioactive molecules in deficient patients.
Hemophilia. This is a family of two major inherited diseases caused by genetic mutations affecting members of the blood clotting cascade. In Hemophilia A the missing protein is Factor VIII and in Hemophilia B – factor IX. Each one of these genes could be incorporated into the genome of host cells thereby providing the essential missing factor.
Adrenal insufficiency. In this type of disease, adrenal cells do not produce sufficient amounts of steroid hormones with a concomitant serious loss of the adrenal-hypophysis-hypothalamic axis. Cortisol replacement is the current state-of-the-art treatment. However, complete cure could be achieved only following implantation of donor or animal adrenocortical cells.
More disorders. While using engineered cells, the βAir platform could be used to treat inherited disorders like ADA (adenosine deaminase) deficiency, PTH (parathyroid hormone) deficiency, GH (growth hormone) deficiency
Stem Cell Development
In the body, stem cells reside in poor-oxygen niches. This is changed during differentiation processes leading to mature cell types. However, current differentiation protocols solely rely on bioactive molecules and level of oxygen does not make any variable. The βAir device allows for a paradigm change, letting researchers to test stem cells differentiation towards any required cells type. Experiments could be done either ‘in-vitro’ or in animal models.
The pig, being a close physiological relative of human beings, is currently developed as a universal organ donor. Immune ‘humanization’ of the pig is in its infancy and time-to-market is counted by many years. However, by using the immune privileged environment created by the βAir device, pig cells and non-vascularized tissues (like pancreatic islets) could be safely used to cure various diseases.
The βAir was recently tested in few pre-clinical studies as a carrier of xenotype cells to cure T1D and adrenal insufficiency at an absence of immunosuppressive drug therapy (see the scientific publication tab).
Laboratory and Pre-clinical Aspects
Oxygen partial pressure is considered ‘normoxic’ when tissue level is about 5% and 21% when ‘in-vitro’ experiments are done. It was a great achievement to discover that pancreatic islets could function under oxygen partial pressure of 60%. The correlation between physiologic functions and oxygen partial pressure is under experimented. The βAir device, variants of which were adopted for rats and pigs, could form an essential tool in such studies.