GeneCellNano Flagship develops new biological drugs, biomarkers and diagnostics for diseases with unmet clinical need. New treatments are developed for cardiovascular diseases and age-related macular degeneration, as well as for brain, ovarian and bladder cancer.
Flagship brings together an interdisciplinary team of dozens of top researchers specialized in biological Advanced Therapy Medicinal Products (ATMP) and nanotechniques linked with their management. Collaborators include clinical actors, leading companies and third sector players.
The cooperation will create a new kind of ecosystem that combines public and private actors and has excellent opportunities to become an international leader in a rapidly developing sector. The Flagship combines innovative basic research, excellent production techniques, long clinical experience and a strong and diverse network of international connections.
The objective of the Flagship is to significantly increase domestic and foreign funding for gene, cell and nanotechnology and to introduce new effective treatments for severe chronic diseases. The aim is also to create new jobs and businesses in the biotechnology and pharmaceutical sectors. This will contribute to the development and economic welfare of Finnish society.
Gene therapy offers a very promising new way to treat several serious diseases by targeting exact causes of the disease or important pathogenetic mechanisms leading to the symptomatic disease. However, this promising technology is still in early development especially in innovation, translating gene therapy to phase I-II trials and large scale GMP manufacturing. Recognized shortcomings include immune responses against the vectors, inability to achieve cell-specific transduction, lack of regulation of the transgene expression and difficulty in targeting transgene integration into so-called safe harbors in human genome. GeneCellNano Gene therapy research line will focus on the above key bottlenecks and will develop state-of the-art program for the creation of next generation ATMPs for chronic diseases.
While the use of T-cells modified with chimeric antigen receptors (CARs) has shown considerable clinical success, several hurdles must still be overcome to enable clinical use of gene-modified NK cells. The cell therapy line of this Flagship focuses on developing more efficient transduction methodologies for NK cells. Second, we will develop and compare two proprietary NK cell processes, one in a closed bioreactor using feeder-free expansion protocols based on cytokine cocktails and another using a proprietary feeder cell line to support expansion, and analyze use of different starting materials for NK cell production. Finally, we will design and test CARs based on new NK-specific signaling domains, assess their efficacy in redirecting NK cell activity in in vitro patient derived solid tumor models and in in vivo experiments.
GeneCellNano Cell therapy research line creates novel solutions for NK cell manufacturing especially in transduction and expansion, and discovers new CAR constructs for controlling NK cell activity, generating a platform for development of new therapeutics.
GeneCellNano Nanotechnology research line aims to develop technology platforms for local and controlled delivery of biologicals to the cardiac, retinal and oncological targets. Platforms are developed for controlled release systems for long-acting, locally applied pharmaceutical products, nanomedicines with improved intracellular disposition, and nanomedicines and extracellular vesicles (EVs) for cell specific delivery properties.