Simone Spuler

Prof. Dr. Simone Spuler is a leading researcher in muscle stem cell biology and gene therapy at the Experimental and Clinical Research Center (ECRC) in Berlin, jointly affiliated with Charité - Universitätsmedizin Berlin and the Max Delbrück Center for Molecular Medicine. She serves as Director of the University Outpatient Clinic for Muscle Diseases, focusing on translational approaches for genetic and age-related muscle disorders.

• Develops CRISPR/Cas9 and base editing therapies for muscular dystrophies (LGMD2A, Dysferlinopathy)
• Translates stem cell research into clinical trials like MuST and BASKet
• Validates GMP-compliant manufacturing of primary human satellite cells (PHSats) for ATMP applications

Her research interests center on muscle stem cell heterogeneity, regenerative medicine, and epigenetic mechanisms in critical illness myopathy. Recent work explores:

• CRISPR editing of patient-derived stem cells
• Humanized mouse models for dystrophy validation
• Epigenetic reprogramming in ICU-acquired weakness
• Decellularization protocols for diaphragm repair

She leads a multidisciplinary team working on:

• Genome editing of CAPN3, LMNA, and Dysferlin mutations
• Stem cell expansion using bacterial nanocellulose
• Development of regenerative therapies for urinary incontinence

Scientific awards include:

• Science4Life Venture Cup 2019 (Pharma Category)
• NLSInvest Award 2022
• Einstein Visiting Fellowship (2019-2022)

Her clinical and entrepreneurial efforts involve founding the startup MyoPax, which develops stem cell therapies for incurable muscle diseases, supported by grants from the German Federal Ministry of Education and Research (BMBF) and Else Kröner-Fresenius Foundation (EKFS). The MuST clinical trial for epispadias patients is funded with €4.3M. Her team collaborates with patient organizations like Coalition to Cure Calpain 3 and Deutsche Gesellschaft für Muskelkranke.