Objective
Drug repurposing can fill an important gap for rare disease patient groups with large unmet medical needs. In comparison to traditional drug development, drug repurposing reduces the time and costs for drug development, regulatory approval, and market authorization. Yet, we need to increase the efficiency of the drug repurposing pathway to provide broader access to new therapeutic modalities for larger groups of patients. SIMPATHIC’s main objective is to accelerate drug repurposing for rare neurological, neurometabolic and neuromuscular disorders. SIMPATHIC’s main accelerating innovation is the simultaneous drug development for groups of patients with different genetic diagnoses but overlapping neurological symptoms and molecular pathomechanisms. SIMPATHIC’s key outputs accelerating the drug repurposing pathway include: Standard operating procedures for culturing stem cell-derived neuronal cell models with proven relevance for clinical symptoms and amenable to high-throughput drug screens; New drug repurposing candidates with proven efficacy in advanced brain-on-a-chip and 3D brain organoid models, as demonstrated by reversal of molecular biomarker signatures and cellular readouts associated with clinical symptoms; Designs of innovative basket clinical trials to which patients with different disorders are recruited, utilizing and aggregating personalized clinical endpoints; A training module for patients and patient organizations to empower them as drivers of the drug repurposing pathway; Blueprints for intellectual property strategies, business models, regulatory dossiers and patient access strategies, developed in co-creation between all relevant stakeholders. SIMPATHIC’s proof-of-concept for the simultaneous development of repurposed drugs for multiple indications will show the path forward to development of personalized treatment opportunities for groups of rare disease patients in a cost- and time-efficient manner.
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HORIZON-RIA - HORIZON Research and Innovation ActionsCoordinator
6525 GA Nijmegen
Netherlands
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Participants (18)
75014 Paris
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72074 Tuebingen
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15341 Agia Paraskevi
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1000 Ljubljana
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1081 HZ Amsterdam
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28002 Madrid
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The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
69120 Heidelberg
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1649-003 Lisboa
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4365 ESCH-SUR-ALZETTE
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59000 Lille
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The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
40225 Dusseldorf
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167 77 Athina
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The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
1081 HV Amsterdam
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75013 Paris
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3743 JN Baarn
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The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
K1H 8L1 OTTAWA
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3584 BK Utrecht
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10117 Berlin
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Partners (2)
Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
Participation ended
19009 Plkermi
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Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
N6 5JW LONDON
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