– Novellus’ patented mRNA-based cell-reprogramming technology creates MSCs with superior immunomodulatory properties
– Novellus’ iPSC-derived MSCs have manufacturing advantages over primary adult, donor-derived MSCs, allowing for larger supply, faster scale-up, and a more consistent cellular product
– MSCs may remedy the cytokine storm that triggers acute respiratory distress syndrome (ARDS) in COVID-19 patients
In a deal inked April 1, 2020, Citius Pharmaceuticals (Nasdaq: CTXR) signed an exclusive six-month option agreement to in-license a stem-cell therapy for acute respiratory distress syndrome (ARDS) from a subsidiary of Novellus, Inc. Headquartered in Cranford, New Jersey, Citius is a specialty pharmaceutical company that develops products internally, as well as in-licenses products related to compelling market opportunities.
Novellus is a pre-clinical stage biotech company in the Cambridge area of Massachusetts that creates engineered cellular medicines. It has a strong intellectual property portfolio (IP) related to RNA gene editing and RNA cell reprogramming that includes more than 30 granted patents.
The relevance of Novellus’s patented process to COVID-19 is that is uses non-immunogenic synthetic mRNA molecules to create induced pluripotent stem cells (iPSCs) that are turned into mesenchymal stem cells (MSCs). MSCs are a cell-type that have demonstrated powerful immunomodulatory properties.
MSCs have taken center stage as a intriguing tool for the treatment of patients with COVID-19 related respiratory complications. This is because there is a foundation of scientific literature and early trials to support the use of MSCs for addressing respiratory disease and distress. Currently, MSCs are being tested in more than 1,000 trials worldwide for diverse range of clinical indications, providing evidence of an acceptable safety profile.
Globally, ARDS is a leading cause of death in severe and critically ill COVID-19 patients. It is a type of respiratory failure in which rapid and severe inflammation damages the patient’s lungs, creating a mortality rate of 30% to 50%.
According to Citius’ CEO, Myron Holubiak:
“ARDS is the most common cause of respiratory failure and mortality in COVID-19 patients. Currently, there is no proven treatment for ARDS. Literature supports the use of counter-inflammatory MSCs for ARDS, and papers published in China have shown that at least seven COVID-19 patients with ARDS responded to MSC therapy. We believe Novellus is at the forefront of creating allogeneic, iPSC-derived MSCs. These cells have the potential to overcome the limitations of MSCs derived from adult donors, which are telomere shortened and introduce variability into the manufacturing process. MSCs have been studied in the treatment of lung injury, and we aim to build upon this work with Novellus’s iPSC-derived MSCs to improve the immunomodulatory response in humans. We have assembled a team of experts who are dedicated to advancing this project to an IND application as quickly as possible.”
According to Novellus’s CSO, Matt Angel, PhD:
“Using our mRNA-based cell-reprogramming technology, Novellus can provide a near-unlimited supply of MSCs for treating patients with ARDS, including those critically ill from COVID-19. These will be allogeneic (‘off-the-shelf’) cells that in vitro have demonstrated much greater expansion potential and much higher immunomodulatory protein expression than donor-derived MSCs. We are excited to employ our technology to such an urgent medical crisis and believe that our MSCs represent an ideal source of cells to be used in this extremely important development effort.”
A Competitor Exploring iPSC-Derived MSCs: Cynata Therapeutics
Another company exploring the use of iPSC-derived MSCs for the treatment of COVID-19 related complications is Cynata Therapeutics, an Australian stem cell company. Cynata’s proprietary technology utilizes iPSCs from an adult donor as the starting material for generating mesenchymoangioblasts (MCAs), which it differentiates in MSCs.
In cases of COVID-19 that produce serious complications, such as acute respiratory distress syndrome, sepsis or cytokine release syndrome, Cynata’s Cymerus™ iPSC-derived MSCs may be positioned to serve an unmet medical need. This is because the company’s iPSC-derived MSCs have demonstrated improvements for these indications in preclinical studies.