StemGen is a clinical-stage biotechnology company focused on developing innovative differentiation biotherapies for solid tissue cancers, particularly glioblastoma multiforme (GBM). Their primary therapeutic approach involves differentiation therapy, which aims to convert cancer stem cells into normal, mature brain cells. This method is particularly significant given the aggressive nature of GBM, which is often deemed incurable with current treatment options. StemGen's lead product, hrBMP4, has received Orphan Drug designation from the European Medicines Agency (EMA) for glioma treatment and has shown promising results in early clinical trials, indicating its potential efficacy and safety in patients with recurrent GBM.

The hrBMP4 protein, part of the TGF-Beta superfamily, operates by binding to specific receptors on GBM cancer stem cells, triggering a cascade of intracellular signals that modify cellular functions. Notably, it activates the onco-suppressor PTEN, which is frequently deleted in GBM cases. The results from a Phase I clinical trial demonstrated that hrBMP4, administered via Convection Enhanced Delivery (CED), was safe and well-tolerated, with some patients experiencing complete or partial tumor responses. StemGen is currently planning confirmatory studies for both recurrent and newly diagnosed GBM, highlighting their commitment to advancing this novel therapeutic approach in oncology.

Italy

StemGen is a clinical stage biotech company developing innovative differentiation biotherapies for cancer. StemGen is also involved in research on neural stem cells.

StemGen

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