This project will develop an innovative therapeutic approach for the treatment of Cystic Fibrosis (CF). This condition originates from the defective function of the CFTR protein, a chloride and bicarbonate permeable transmembrane channel. This project will evaluate small molecules capable of facilitating the transmembrane transport of anions such as chloride and bicarbonate and will thus enable CF treatment by replacing the missing CFTR anion permeation activity. This represents an unexplored path in the treatment of CF and a paradigm shift with respect to current strategies searching for a cure for CF. Instead of focusing on the development of mutation-specific treatments, we plan to develop a therapy applicable to CF patients, regardless of the type of mutation they harbor. Thus, this therapeutic approach overcomes the limitation of current mutation-specific treatments and is applicable to CF patients in general. To achieve this goal we have set up a comprehensive program to validate a research concept and complete the preclinical development of a new lead compound, making it ready for early clinical development. A rmultidisciplinary team of qualified researchers have been assembled to bring to conclusion a truly translational project from the synthesis of new compounds to validation on animal models. Cystic Fibrosis affects more people than any other rare disease. Therefore, it could be said, at least in quantitative terms, that CF qualifies as the main target of the topic. This project aims to complete the preclinical development of novel, innovative drugs based on a radically new concept in Cystic Fibrosis therapies. This result fully addresses the expected impact set out in the work programme of advancing the development of new therapeutic options for patients living with rare diseases as well as contributing to reach the IRDiRC objective to deliver 200 new therapies for rare diseases by 2020.
Nanoparticles and new drugs are undergoing complex toxicity evaluation prior to their use in products for customers. However, the traditional pre-clinical testing protocols are delivering significant disadvantages both from ethical (animal testing) and functional (high attrition rate in clinical testing) point of view. ActiTOX reacts to this problem by the development of a novel, pre-clinical, high content, in vitro testing platform focused on the evaluation of nanoparticles/drugs via ADME (absorption, distribution, metabolism, excretion) toxicity studies. In order to advance the current state of art the project will employ biomimetic scaffolds, drug delivery systems, human induced pluripotent stem cells (iPSCs), 3D-microfluidics and modular bioreactor technology. ActiTOX aims to increase the relevance of in vitro studies and provide a scalable approach for their use in drug development and toxicological screening. In order to achieve the project goals, a training network enabling efficient short-term secondments of ER and ESR between academia and industry will be established. The cooperation between stakeholders helps closing the gap between them and oint innovation to foster the current state of the art. ActiTOX will mediate it via networking, research, training, workshop, innovation and dissemination actions in form of short-term secondments. The project fulfils the aim of a RISE project and delivers significant social impact, increases knowledge based economy and creates long lasting cooperation strengthening the European research area.
iP-OSTEO project focuses on development of novel cell-based scaffolds for bone and cartilage repair in patients with poor regenerative capacity. We are proposing technology combining iPSCs with improved regeneration capacity combined with nanostructured scaffolds based on electrospun scaffolds and drug delivery system stimulating cell regeneration based on electrosprayed and spray-dried particles. The system will deliver novel treatment method for older patients, for which the current cellular and cell-free methods are ineffective. iP-OSTEO will reach its goals by creating an international and interdisciplinary training program. 7 companies (SMEs) and 7 academic institutions across European Union will join there forces though dedicated secondments. The activities will involve networking, research/training, workshop and dissemination secondments. The project has in total 239 secondments involving exchange of Early-stage and Experienced researchers. The consortium is bonded by Dr. Eva Filova – young scientist with experience in bone and cartilage tissue engineering. The iP-OSTEO project will help in better integration of academic and industrial stakeholders across Europe and help diseased people by providing novel therapeutic methods. Nevertheless, project has 169 ESR secondment months leading to training of new generation of scientists with international connection and knowledge of intrasectorial enviroment.
EBiSC2 builds on the achievements of the European Bank for iPSCs (EBiSC1) in centralising existing capacities across Europe in a unique banking and distribution infrastructure for research use in response to the increasing demand for human induced pluripotent stem cells (iPSC). Significant progress towards this aim has been made by EBiSC1; further resources, however, are required to ensure self-sustainability. Key partners of EBiSC1 who have delivered major assets of the current bank, join efforts to establish EBiSC2 as self-sustainable, central bank. Based on a gap analysis of the EBiSC1 endeavours towards sustainability, and focussing on user demand, scientific excellence and productivity, EBiSC2 will deploy a business strategy for a sustainable, non-for-profit bank providing access to disease-relevant and quality-controlled iPSCs, along with comprehensive data and freedom to operate for academic and commercial use. To meet evolving requirements from industry and academia, the cell catalogue will be constantly enriched through on-demand generation of new iPSC lines, including gene-edited lines and isogenic controls, iPSC-derived and progenitor cells. EBiSC2 will distribute cell lines and develop a range of additional cell services (incl. screening panels of disease-relevant iPSC and control lines in ready-to-use-formats) to extend its offer, while reducing operational costs through state-of-the-art upscaling and automation enabling bulk production of standardised high-quality cells. Proof-of-concept studies performed jointly by academia and industry will demonstrate the reliability and robustness of the lines for disease modelling and screening and enrich the EBiSC2 catalogue with extensive data. To bundle resources, EBiSC2 focuses on collaboration with iPSC programmes and aims to serve as central hub for EU-funded projects to bank their iPSC lines, and thus, enable long-term access by the research community to the results of European investments.