Diabetes mellitus type 1 (DM1) represents up to 10% of all diabetes cases. The disease can occur at any age, but the onset is more common in children and young adults. It is estimated that worldwide approximately 86,000 of new cases of DM1 are diagnosed in children under the age of 15 each year, and the incidence is increasing by approximately 3% annually (IDF Diabetes Atlas, 2015).
The most important factor underlying the etiology of DM1 is impairment in the regulation of the immune system, associated with a T cell dependent autoimmune attack. In diabetic patients insulin-producing β cells are recognized and destroyed by self-immune cells. Despite the fact that we are currently able to detect DM1 before complete loss of insulin-producing cells and to identify individuals being in the asymptomatic stage of DM1 development (presence of autoantibodies against β cell antigens), there are presently no available effective therapies to prevent disease progression and clinical manifestation. The current medical management for DM1 is based on insulin replacement therapy. The aim is to lower fasting blood glucose levels as much as possible to the normal range of 3.9-5.5 mmol/L. Good glycemic control helps to avoid acute episodes of life-threatening hyper- and hypoglycemia, as well as long-term neurological dysfunctions and vascular complications, leading to severe disability (e.g. vision loss, urinary incontinence, loss of feeling in limbs, muscle weakness and pain, and end-stage kidney disease).
The overall objective of the project was to carry out a set of actions leading to a feasibility report on full-scale market commercialization. The project was aimed at de-risking the investment in an innovative project and a company for investors. Phase 1 proved to be instrumental in the Company’s understanding the regulatory and business environment. The company has performed a thorough investigation of many assumptions which in the end allowed for an evolution of the business idea, that attracted both VC and PE investors. The Company has identified key market barriers, which are of regulatory nature and analyzed the market to validate the above-the-standard effect of the therapy and influence on the national healthcare (including possible reimbursement by governments with relation to the overall costs of the diabetes care). The main aim for PolTREG was to increase clinical availability of vaccine using autologous TREGs for the benefit of the patient and design of cost-effective process thus increase likelihood of market uptake and distribution of innovative (better targeted) therapy developed by an European SME.
