Project description
Tailored antisense oligonucleotide treatments for ultra-rare diseases
Modern medicine’s goal is to customise diagnoses and treatments for individual patients, particularly those lacking available therapies due to existing development paradigms. The MSCA-funded MMM project aims to create personalised antisense oligonucleotide (ASO) treatments for patients with ultra-rare disease mutations, incorporating omics-based theranostics, preclinical target validation, innovative trial methodologies and an ethical framework for personalised genetic therapies in accordance with European Medicines Agency guidelines. Given that this emerging scientific field necessitates a fresh cohort of open-minded, technology-fluent and application-oriented specialists, the project provides driven doctoral candidates with an opportunity to construct and undergo training across all fundamental modules essential for tailored ASO treatments. This endeavour imparts education to future experts in drug development and translational processes.
Objective
Modern medicine seeks to personalize diagnosis and treatment to the needs of individual patients. This need is heightened in particular for those individual patients where no treatments are available – and where current standard treatment development paradigms exclude to generate such. The ultimate goal of Medicine made to Measure (MMM) is to go even beyond the latest genetic medicine approaches, implementing a novel paradigm of treatment development: the field of single patient tailored antisense oligonucleotide (ASO) treatments for patients with nano-rare disease mutations. Specifically, MMM will develop core modules of a novel “out-of-the-box”, but still EMA-advised treatment development path for these patients - from omics-based theranostics, via preclinical target validation to innovative trial methodology and a pioneering ethical framework for individualised genetic therapies. MMM provides a unique opportunity for doctoral candidates to be involved in building - and to receive training - in all core modules needed for tailored ASO treatment in Europe. This new scientific field requires a new generation of open-minded, technology-fluent and applications-oriented experts.
MMM will help educate future drug development and translational experts in adopting and disseminating such a cross-specialty approach. Our idea for this innovative training network is built on our personal experience and understanding of the importance of single patient tailored genetic treatments in the years to come. We propose a consortium of world-leading experts and partners that present with extended experience and relevant know-how in molecular biology, neurology, translational medicine, biochemistry, bioinformatics, philosophy, mathematics, regulatory science and engineering. Thus, MMM provides an exceptional platform to young, ambitious and talented researchers who like to engage in supporting the concept of single patient tailored treatments.
Fields of science (EuroSciVoc)
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques.
- natural sciencesbiological sciencesbiochemistry
- natural sciencesbiological sciencesgeneticsmutation
- medical and health sciencesbasic medicineneurology
- humanitiesphilosophy, ethics and religionphilosophy
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Keywords
Programme(s)
- HORIZON.1.2 - Marie Skłodowska-Curie Actions (MSCA) Main Programme
Funding Scheme
HORIZON-TMA-MSCA-DN - HORIZON TMA MSCA Doctoral NetworksCoordinator
72074 Tuebingen
Germany
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Participants (7)
2333 ZA Leiden
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07546 Gera
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80539 MUNCHEN
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2100 Goedoello
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The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
40127 Bologna
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The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
751 05 Uppsala
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69120 Heidelberg
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Partners (14)
Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
02125 Boston
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The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
66117 Saarbrucken
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Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
151 85 Sodertaelje
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Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
75014 Paris
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Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
72770 Reutlingen
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Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
5231 DD 'S-Hertogenbosch
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Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
72076 Tubingen
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The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
72072 Tübingen
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Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
98693 Ilmenau
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Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
40126 Bologna
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Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
2100 Godollo
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Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
45147 Essen
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Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
3531AH Utrecht
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Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
CB2 1TN Cambridge
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