Cardiovascular diseases including myocardial infarction (MI), which entails the irreversible loss of heart muscle tissue, constitute a major socio-economic burden in global healthcare. With whole organ transplantation as the only treatment option for end-stage heart failure, MI patients could particularly benefit from advanced cell therapies aimed at the functional reconstitution of damaged hearts. Human induced pluripotent stem cells (hiPSCs) can be derived by reprogramming patients’ somatic cells. In contrast to adult (stem) cells e.g. from blood, bone marrow or the heart, hiPSCs have unlimited expandability and differentiation potential into all relevant cell types including cardiomyocytes, endothelial cells, pericytes and connective tissue-forming cells, making them highly attractive as a universal cell source for organ repair. However, technologies for the robust therapeutic scale production of hiPSC-derived progenies in line with GMP standards and at reasonable cost are currently lacking. TECHNOBEAT’s ultimate objectives are 1) to advance therapeutic scale cell production through innovative bioreactor technologies and novel cell monitoring tools, and 2) to develop regulatory compliant bioprocessing of innovative iPSC-based cardiac µ-tissue. The clinical translation of cardiac µ-tissue will require 3) the development and application of tools for improved cell delivery and longitudinal in vivo monitoring of cell grafts, and 4) proof-of-concept for safety and functional integration in physiologically relevant preclinical models of cellular heart repair. Through its interdisciplinary excellence, TECHNOBEAT’s consortium of leading European stem cell researchers, clinicians, tissue-, bioprocess-, and technical- engineers in industry and academia is ideally positioned to address these ambitious objectives. It will provide new treatment options for suffering patients and increase Europe’s attractiveness as a hub for innovative medical technologies.

Stress Urinary Incontinence (SUI) is a disease affecting over 200 million people worldwide. It represents a condition with a prevalence of 20-50% in women, thereby creating an immense socio-economic burden. The currently available treatment strategies entail various complications and offer only short-term relief to the patients. Tissue engineering using autologous cells offers a feasible alternative for functional restoration of the damaged urinary sphincter muscle and represents an ideal treatment option that could reverse the underlying pathologic conditions. MUSIC aims at translating basic knowledge on regenerative medicine (RM) and stem cell therapy into the clinic by undertaking a "first-in-man" multisystem study using autologous muscle precursor cells (MPCs) in a combination with neuromuscular electromagnetic stimulation (NMES) in 40 female patients. We will carry out the specific tasks to prove safety and efficacy of the proposed novel multilevel treatment as well as reproducibility of the therapeutic effect. Additional objectives are optimization of the advanced-therapy medicinal product (ATMP) towards totally xeno-free and facilitated manufacturing as well as the introduction of a novel injection technique for more efficient and precise implantation of the final product. Combining expertise, MUSIC features a unique infrastructure, including the knowledge of experts in the fields of RM, urology, cellular biology and biomaterials throughout Europe (CH, NL, UK, A, D). The MUSIC consortium has an exclusive opportunity to determine the validity of this MPC cellular treatment in combination with NMES and to further improve its feasibility and clinical efficacy. The ultimate goal is to significantly improve the patients` quality of life and to exploit a future commercial opportunity by expanding the know-how to various smaller RM centers and companies within Europe, thus, making personalized medicine using autologous cells a more feasible SUI treatment option.

Parkinson’s Disease (PD) is a major neurodegenerative disorder with no established treatment modalities capable of modifying disease pathology, and no means of early diagnosis. Vaccines targeting aSyn aggregates are a promising route to disease-modifying therapy for PD, but the current generation of PD vaccines utilise conventional formulations, which are limited in their immunogenicity and require substantial quantities of adjuvant to achieve efficacy. NEXGEN’s proprietary WISIT vaccine platform is the first of the novel class of gluconeoconjugate vaccines (GNCVs), which are administered intradermally and specifically formulated to leverage skin dendritic cells (DCs) to generate substantially stronger and more specific immune responses than conventional vaccines. These stronger immune responses allow substantial reduction in adjuvants, while simultaneously increasing therapy efficacy. NEXGEN will identify and characterise candidate WISIT constructs targeting aSyn (PD-WISITs) and develop a novel extracellular vesicle (EV)-based biomarker assay that enables early diagnosis of PD using liquid biopsies, suitable for point of care use. Safety and efficacy of PD-WISITs will be demonstrated preclinically, before being translated to first-in-human Phase I/Ib clinical trials, along with the novel EV-based biomarker assay. The results of NEXGEN will be the extraordinary accomplishments of cheap and effective disease-modifying treatment of early PD and a novel biomarker assay to diagnose and guide prodromal/early PD treatment. Further still, GNCV technology will be clinically demonstrated, which has the potential to be transformative to the treatment of a wide range of additional diseases, resulting in far-reaching impacts to the health of millions.