About us
The Project
The central nervous system has a limited capacity for self-repair. Therefore, technologies to replace lost neurons after an injury, including stroke or neurodegenerative diseases, are of great need. The project REGENERAR aims to develop a non-viral delivery formulation up to TRL4 for the delivery of a brain reprogramming formulation.
In the first step, the safety and targeting of the formulation will be tested in vitro.
In the second step, the in vivo safety, elimination, targeting and cell reprogramming will be evaluated. In addition, toxicological studies will be performed in GLP conditions to evaluate both in vivo systemic and local (brain) effects of the formulation.
The development of the non-viral formulation will take into account the contributions of key stakeholders in the area of gene editing, health institutions, and patient associations, among others. A roadmap will be developed for upscaling innovation to higher TRL.
Objectives
- Development of a non-viral formulation for brain cell reprogramming
- Proof-of-concept demonstration of brain cell reprogramming both in vitro and in vivo
- Advancement of knowledge and education in cellular reprogramming
In progress
Work Packages
REGENERAR WP1 will focus on the development and characterization of innovative non-viral formulation for brain cell reprogramming.
REGENERAR WP2 will focus on in vitro demonstration of safety, targeting and efficiency of the innovative non-viral formulations for brain cell reprogramming.
REGENERAR WP3 will study the best route of administration of the innovative non-viral formulations to achieve effective brain delivery.
REGENERAR WP4 will evaluate in vivo the efficiency of the innovative non-viral formulations for brain cell reprogramming.
REGENERAR WP5 will focus on the communication, dissemination and exploitation of project achievements.
REGENERAR WP6 focus on project coordination and management to ensure project’s objectives are achieved and that the Project is implemented in compliance with the EC Grant Agreement (GA) and Consortium Agreement (CA).
Team
The consortium includes six partners from three European Member states, including 2 academic institutions (UC, Portugal and HMGC, Germany), 1 research institute (Fraunhofer Institute, Germany), 1 biotech SME (SINGLE, Sweden), 1 large company (Eunice Costa, Hovione, Portugal) and 1 project management company (SPI, Portugal).
Consortium partners have expertise in different fields, including brain drug delivery protein production and epigenome editing, production of nanoparticles under GLP conditions, spatially resolved transcriptomics, and safety and toxicity studies.
Coordinator
Universidade de Coimbra (UC)
The research group has two main avenues of research:
- Disease/ageing modeling and drug screening program
- Regenerative medicine, particularly focused in regeneration mediated by controlled delivery of drugs.
The research group uses many tools to accomplish this goal, including the design of new biomaterials with relevant biological information, molecular and cell biology, microfluidic systems, high content analysis, and animal experimentation. Nanomedicine platforms to modulate the activity of stem cells and their progenies. The development of a wide spectrum of nanotechnologies (referred as Nanomedicine by National Institutes of Health for applications in the biomedical area) during the last years are very promising for the study of stem cell biology and to control exogenous and endogenous stem cells for regenerative medicine. The group is particularly interested to use these tools to induce in vivo stem cell differentiation and to mobilize stem cells from their niches to treat cardiovascular diseases. For this purpose, they are developing nanomaterials that release efficiently biomolecules (small molecules, proteins or non-coding RNAs) to manipulate stem cells or their progenies.
Partners
Sociedade Portuguesa da Inovação (SPI)
Since its inception, SPI has become a leading promoter of linkages between private sector companies, Science and Technology institutions, and National and International public and private organizations. From its onset, SPI embraced the mission of managing projects that promote regional, national and international development, stimulate entrepreneurship, innovation and knowledge management and encourage internationalization and creation of strategic partnerships and business relationships.
SPI has established a unique in-house capability that combines its knowledge of Innovation, Science and Technology with its fundamental skills in territorial planning and urban development. Since its creation in 1997, SPI was involved in the Leadership/Partnership in about 25 FP7 projects, 50 Horizon 2020 projects and 28 Horizon Europe projects and collaborated with more than 80 districts in Portugal, including setting local multi-actor forums.
Helmholtz Munich (HMGU)
Headquartered in Neuherberg in the north of Munich is the largest scientific organization in Germany with more than 40,000 employees at 19 research centers.
The Epigenetic Engineering group, lead by Prof. Dr. Stefan H. Stricker, investigate which mechanisms ensure that cells do not change identity during the lifetime and how we can manipulate those to freely convert cell fates. We employ a wide range of CRISPR methods to test in vitro and in vivo, which epigenetic marks and gene activities have functional relevance in mediating cell identity or disease phenotypes in the brain.
Hovione Farmaciência S.A (HOVIONE)
Over 60 years of experience in the development and compliant manufacture of Active Pharmaceutical Ingredients and Drug Product Intermediates. With four FDA inspected sites, USA, China, Ireland and Portugal, Hovione offers branded pharmaceutical customers services for the development and compliant manufacture of innovative new drugs including highly potent compounds. For generic pharmaceutical customers the company offers niche generic API products. In the inhalation area, Hovione is the only independent company offering a complete range of services. Hovione is a member of Rx-360, EFCG and participates actively in industry quality improvement initiatives to lead new global industry standards. Hovione is a company with a culture based on innovation, quality and delivery.
Single Technologies AB (SINGLE)
Single Technologies is pushing the limits of genomics by combining single molecule imaging, fast large area confocal scanning, grating techniques, fluidics, nanotechnology. Our technology is being explored by leaders and industry in the genomics field who cares about Big Data generation.
Single Technologies was founded out of KTH research in single molecule imaging and biotechnology. Our prediction is an exponential increase of the need of sequenced data to enable the full potential of applying Big Data and AI to genetic analysis, in particular accurate diagnoses and personalized treatments, where Singe Technologie’s breakthroughs will play a major role enabling such progress.
Fraunhofer-Gesellschaft (Fraunhofer ITEM)
Currently the company operates 76 institutes and research units throughout Germany.
The Department of Inhalation Toxicology, lead by Dr Gustav Bruer offers a broad range of toxicological tests enabling assessment of potential risks to human health. Substances investigated include chemicals, particles, complex mixtures, and nanomaterials. The focus is on inhalation toxicology and on characterizing inhalable substances. Expertise ranges from the development and implementation of testing strategies to accompanying and providing consultancy to companies in registering their products. A sound basis for their work is provided by the institute’s expertise in inhalation, fiber and particle toxicology, and in aerosol research and chemical analysis.