ProjectSIMPATHIC – Accelerating drug repurposing for rare neurological, neurometabolic and neuromuscular disorders…

Basic data

Accelerating drug repurposing for rare neurological, neurometabolic and neuromuscular disorders byexploiting SIMilarities in clinical and molecular PATHology
01/06/2023 to 31/05/2028
Abstract / short description:
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 cellderived
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.

Involved staff


Faculty of Medicine
University of Tübingen

Contact persons

Faculty of Medicine
University of Tübingen

Other staff

Faculty of Medicine
University of Tübingen
Universität Tübingen

Local organizational units

Institute of Medical Genetics and Applied Genomics
Department of Diagnostic Laboratory Medicine
Hospitals and clinical institutes, Faculty of Medicine


Brüssel, Belgium

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