Summary
Rare diseases, though individually infrequent, collectively pose important challenges for patients, clinicians and researchers in terms of diagnostics, healthcare and treatment. Myotonic Dystrophy (DM), the most predominant inherited muscular dystrophy in adults, impacts 60,000-70,000 individuals in Europe. Its complex genetic and clinical variability result in a lack of robust genotype-phenotype correlations, which complicates the understanding of tissue-specific disease mechanisms, the development of effective therapies and the stratification of patients into well-defined clinical groups for clinical trial purposes. In recent years, Antisense Oligonucleotides (ASO) have emerged as promising therapeutics, notably in neuromuscular disorders. However, previous clinical trials in DM have failed due the reduced efficacy and bioavailability of the ASO tested.
ENTRY-DM aims to train 14 DCs in translational research, through the combination of basic and clinical competencies across multiple disciplines, as well as strong soft and transferable skills. Our interdisciplinary network seeks to enhance scientific and technological knowledge, spanning from disease mechanisms, ASO design and delivery strategies, to clinical trial preparedness, using innovative multidisciplinary approaches and best practices. The consortium includes experts in DM research, bioengineering of model systems for preclinical drug screening, ASO chemistry, as well as clinical and neuropsychological assessment. Close collaborations with multi-sectoral partners will address the challenges in technology transfer, providing high-quality bench-to-bedside training to the next generation of researchers.
Through these efforts, ENTRY-DM will catalyze ASO therapeutic development towards upcoming impeding clinical trials, establishing solid foundations for future clinical applications, increased investment and entrepreneurship ventures in the field of DM and other related diseases.
ENTRY-DM aims to train 14 DCs in translational research, through the combination of basic and clinical competencies across multiple disciplines, as well as strong soft and transferable skills. Our interdisciplinary network seeks to enhance scientific and technological knowledge, spanning from disease mechanisms, ASO design and delivery strategies, to clinical trial preparedness, using innovative multidisciplinary approaches and best practices. The consortium includes experts in DM research, bioengineering of model systems for preclinical drug screening, ASO chemistry, as well as clinical and neuropsychological assessment. Close collaborations with multi-sectoral partners will address the challenges in technology transfer, providing high-quality bench-to-bedside training to the next generation of researchers.
Through these efforts, ENTRY-DM will catalyze ASO therapeutic development towards upcoming impeding clinical trials, establishing solid foundations for future clinical applications, increased investment and entrepreneurship ventures in the field of DM and other related diseases.
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More information & hyperlinks
| Web resources: | https://cordis.europa.eu/project/id/101169266 |
| Start date: | 01-03-2025 |
| End date: | 28-02-2029 |
| Total budget - Public funding: | - 3 693 326,00 Euro |
Cordis data
Original description
Rare diseases, though individually infrequent, collectively pose important challenges for patients, clinicians and researchers in terms of diagnostics, healthcare and treatment. Myotonic Dystrophy (DM), the most predominant inherited muscular dystrophy in adults, impacts 60,000-70,000 individuals in Europe. Its complex genetic and clinical variability result in a lack of robust genotype-phenotype correlations, which complicates the understanding of tissue-specific disease mechanisms, the development of effective therapies and the stratification of patients into well-defined clinical groups for clinical trial purposes. In recent years, Antisense Oligonucleotides (ASO) have emerged as promising therapeutics, notably in neuromuscular disorders. However, previous clinical trials in DM have failed due the reduced efficacy and bioavailability of the ASO tested.ENTRY-DM aims to train 14 DCs in translational research, through the combination of basic and clinical competencies across multiple disciplines, as well as strong soft and transferable skills. Our interdisciplinary network seeks to enhance scientific and technological knowledge, spanning from disease mechanisms, ASO design and delivery strategies, to clinical trial preparedness, using innovative multidisciplinary approaches and best practices. The consortium includes experts in DM research, bioengineering of model systems for preclinical drug screening, ASO chemistry, as well as clinical and neuropsychological assessment. Close collaborations with multi-sectoral partners will address the challenges in technology transfer, providing high-quality bench-to-bedside training to the next generation of researchers.
Through these efforts, ENTRY-DM will catalyze ASO therapeutic development towards upcoming impeding clinical trials, establishing solid foundations for future clinical applications, increased investment and entrepreneurship ventures in the field of DM and other related diseases.
Status
SIGNEDCall topic
HORIZON-MSCA-2023-DN-01-01Update Date
23-12-2024
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Organisations
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| INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (Coördinator)
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| AGENCIA ESTATAL CONSEJO SUPERIOR DE INVESTIGACIONES CIENTIFICAS
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| ARTHEX BIOTECH S.L.
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| ASSOCIATION INSTITUT DE MYOLOGIE
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| BIOBAM BIOINFORMATICS SL
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| CENTRE D'ETUDE DES CELLULES SOUCHES
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| EATRIS ERIC
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| ECRIN EUROPEAN CLINICAL RESEARCH INFRASTRUCTURE NETWORK
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| EURO-DYMA
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| FUNDACIO INSTITUT DE BIOENGINYERIA DE CATALUNYA (IBEC)
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| GENARTIS SRL
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| GOETEBORGS UNIVERSITET
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| LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN
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| Myotonic Dystrophy Foundation
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| SORBONNE UNIVERSITE (SU)