PHSat manufacture

Our core technology is our patented method for the isolation and cultivation of regenerative muscle stem cells.

This enables us to produce large high-purity populations of stem cells that we call PHSats: Primary Human Satellite cell derived muscle stem cells. In contrast to conventionally cultured muscle cells, PHSats retain the regenerative capacity of satellite cells.

From just a few grammes of tissue, more than 100 million pharmaceutical quality PHSats can be produced. In preclinical studies PHSats built new muscle tissue and repopulated the stem cell niche, thereby providing both short and long-term regenerative effects.

In patients with local muscle defects, this technology enables effective autologous treatment.

© Eric Metzler
Related Publications
Muscle stem cell engineering from iPSCs

The PHSats phenotype is our target for cell engineering. Now we are engineering PHSats-analogs that meet disease-specific needs.

Induced pluripotent stem cells (iPSCs) are precursor cells that can differentiate into muscle stem cells. Their ubiquity makes them ideal for cell engineering. From iPSCs we are engineering PHSats-analogs that are universally immunologically tolerable and can be used for allogenic therapies.

Our engineered PHSats will be available as “off-the-shelf” products for the treatment of acute muscle atrophy.

Related Publications
mRNA-delivered gene repair

The delivery of genetically repaired PHSats will be a groundbreaking development in the treatment of muscular dystrophies providing both immediate and long-term regeneration of functionally critical muscles.

We have developed mutation specific gene-editing toolkits based on CRISPR/Cas technology that ensure precise, efficient and safe repair of mutations in genes encoding therapeutically relevant muscle proteins. When combining our patented PHSats expansion process with GMP-compliant mRNA delivery of the repair toolkit, the patient’s own stem cells can be cured.

© Andreas Marg
Related Publications
Behaviour of muscle stem cells in conventional culture

Satellite cells are the true stem cells of skeletal muscle and are essential for growth, maintenance, and regeneration of the tissue.

When extracted from their local niche in muscle and cultivated using conventional methods, these cells quickly differentiate and fuse into myotubes (the precursors of myofibers), thereby losing their regenerative and proliferative characteristics. Conventional culture methods can also lead to the satellite cells beginning as a minority population amongst other co-isolated cell types and being quickly overgrown. Therefore, conventional culture methods are unsuitable for pharmaceutical expansion processes since the resultant populations lack the purity and potency essential for therapeutic efficacy.

Additional reading
Related publications Muscle Research Group, Charité & MDC