The world’s first exosome therapy for osteoarthritis (OA) to achieve clinical proof of principle. It is a ready-to-use ATMP with demonstrated results for addressing cartilage loss and inflammation in OA.
Exosome Therapy for Osteoarthritis
11,000
Million USD, Global OA Therapeutics Market by 2025
23.4
CAGR Exosome Therapeutics Market for the 2022-2030 period
32.5
Millions of adults OA Prevalence in the US
“The exceptional results of the first patient reassured our confidence in this product’s potential to become an unprecedented disease-modifying treatment for osteoarthritis, which affects the quality of life of more than 500 million patients around the world. This milestone positioned C4C’s exosome therapy at the forefront of the field, and, to capitalize upon it, we decided that forming a new venture was the best route for clinical and financial success.”
Maroun Khoury, Ph.D., Chief Scientific Officer
Small extracellular vesicles from our proprietary allogeneic stem cells. Understanding the potential of Mesenchymal Stem Cells-derived exosomes as both biotherapeutics and drug/gene delivery systems to treat osteoarthritis patients, among other applications, C4C protocolized the GMP production of exosomes isolated from its proprietary allogeneic umbilical cord-derived stem cells, considered the youngest and fittest source of stem cells. Several CMC studies confirmed that batches of the exosomal therapy are reproducible in size, concentration, and biomarkers. Moreover, we demonstrated that these exosomes purified from umbilical cord mesenchymal stem cells (UC-MSCs) present a distinctive and reproducible miRNA signature across different donors. The uniqueness of the resulting, purified exosomes obtained from these processes encouraged the filing of a new patent that aims to secure our competitive edge in the nanobiologics field.
Extensively studied exosome therapy for osteoarthritis. Cells for Cells has conducted more than a dozen IND-enabling studies with its UC-MSC-derived exosomes to understand their therapeutic action. These comprehensive studies ranged from the characterization of exosomes’ parental cells to potency tests and clinical-grade product characterization. The results from such research confirmed that exogenous small extracellular vesicles (sEVs) from UC-MSCs are effectively internalized by chondrocytes and favor macrophages’ M2 anti-inflammatory phenotype. In addition, exosomes remained in the local environment after intraarticular injection in a biodistribution assay and reduced cartilage damage associated with OA in preclinical models of collagenase-induced OA. The sum of the results extensively explained and supported the therapeutic potential of our pioneering exosome therapy.
First in the race to the clinic. In 2021, Cells for Cells dosed the world’s first-ever patient with an exosome-produced therapy for joint disease. Notably, the first patient experienced a significant improvement in life quality and unprecedented WOMAC score reduction only six months after a single intra-articular administration. The lack of any observed adverse effect in this first-in-human trial reinforced the safety profile of C4C’s MSC-derived exosomes and their potential as disease-modifying agents. These results were presented at ISCT 2022 in San Francisco and the ISEV 2022 meeting in Lyon, France. More recently, Cells for Cells became the first company to initiate a Phase I trial (the Exo-OA1 trial) with an exosome-produced therapy for osteoarthritis. Following these milestones, the company is tech-transferring its enabled exosome platform to initiate clinical trials in the US.
