MyoPax
MyoPax is a pioneering biotech company dedicated to developing effective muscle regeneration therapies for muscle disorders. Originating from the Muscle Research Unit of Charité Universitätsmedizin and the Max-Delbrück-Center in Berlin, it aims to become an international leader in biological muscle therapy by leveraging advanced stem cell manufacturing, cell engineering, and gene-editing technologies. The company focuses on restoring muscle function in local defects and wasting disorders through proprietary technologies such as PHSats, iPSC-derived muscle precursors, and gene repair tools. MyoPax is actively involved in clinical trials, collaborations, and has received recognition and funding from various institutions including the FDA, Bayer, and the European Union.
Industries
Nr. of Employees
small (1-50)
Products
Autologous expanded muscle stem cell therapy for local muscle defects
A patient-specific cell therapy produced by isolation and expansion of a patient’s own muscle satellite cells and delivered in a ready-to-use formulation for repair of local muscle defects.
Allogeneic engineered iPSC-derived muscle precursor cell therapy (off-the-shelf)
Engineered muscle precursor cells derived from induced pluripotent stem cells and modified for reduced immunogenicity to provide an off-the-shelf therapeutic for acute muscle wasting conditions.
Autologous gene-corrected muscle stem cell therapy for genetic muscular dystrophies
Patient-derived muscle stem cells in which disease-causing mutations are repaired using precise gene-editing toolkits, followed by expansion and targeted injection into affected muscles to restore healthy tissue.
Autologous expanded muscle stem cell therapy for local muscle defects
A patient-specific cell therapy produced by isolation and expansion of a patient’s own muscle satellite cells and delivered in a ready-to-use formulation for repair of local muscle defects.
Allogeneic engineered iPSC-derived muscle precursor cell therapy (off-the-shelf)
Engineered muscle precursor cells derived from induced pluripotent stem cells and modified for reduced immunogenicity to provide an off-the-shelf therapeutic for acute muscle wasting conditions.
Autologous gene-corrected muscle stem cell therapy for genetic muscular dystrophies
Patient-derived muscle stem cells in which disease-causing mutations are repaired using precise gene-editing toolkits, followed by expansion and targeted injection into affected muscles to restore healthy tissue.
Services
Clinical and regulatory development support for early-phase cell and gene therapy trials
Support for translational planning, early-phase clinical trial preparation, and regulatory interactions for cell- and gene-based muscle therapies.
GMP-aligned manufacturing and supply of cell therapy product batches
Manufacturing processes intended to produce pharmaceutical-quality muscle stem cell products in patient-specific and non-patient-specific batches for clinical use.
Clinical and regulatory development support for early-phase cell and gene therapy trials
Support for translational planning, early-phase clinical trial preparation, and regulatory interactions for cell- and gene-based muscle therapies.
GMP-aligned manufacturing and supply of cell therapy product batches
Manufacturing processes intended to produce pharmaceutical-quality muscle stem cell products in patient-specific and non-patient-specific batches for clinical use.
Expertise Areas
- Cell therapy manufacturing and scale-up
- Induced pluripotent stem cell (iPSC) engineering
- Gene editing for monogenic muscle disorders
- Preclinical in vivo model development
Key Technologies
- Primary human muscle satellite cell isolation and expansion
- Induced pluripotent stem cell (iPSC) differentiation to myogenic lineage
- CRISPR/Cas gene editing (including base editing and single-cut methods)
- mRNA-mediated delivery of gene-editing toolkits