Next-generation engineered stem cell platform of induced mesenchymal stem cells (i-MSCs)

Citius is developing a unique, proprietary stem cell platform for the treatment of respiratory conditions associated with acute inflammation, with an initial indication in the treatment of acute respiratory distress syndrome (ARDS).

Citius' i-MSCs are derived from iPSCs originating from a qualified single-donor dermal fibroblast, resulting in one homogeneous, validated source for all future cells. A patented synthetic, non-immunogenic mRNA (non-viral) high efficiency cell reprogramming technique is applied and expanded under cGMP guidelines to create a clonal iPSC Master Cell Bank. The i-MSCs produced from this clonal technique are differentiated from human donor-derived MSCs (bone marrow, placenta, umbilical cord, adipose tissue, or dental pulp) by providing genetic homogeneity. In vitro studies showed that Citius’s i-MSCs exhibit superior potency and high cell viability. Moreover, i-MSCs secrete immunomodulatory proteins that may reduce or prevent pulmonary symptoms associated with ARDS. Citius believes that the characteristics of its single-donor i-MSCs support clonal production at scale with consistent quality and greater potency, and may offer patients and clinicians a promising treatment option for ARDS.

Program Highlights

  • Novel stem cell therapy for the treatment of acute inflammatory respiratory disorders including acute respiratory distress syndrome (ARDS). Our stem cell therapy is in the pre-clinical stage.  The results of our pre-clinical studies will determine whether we seek regulatory approval for the therapy. 
  • i-MSCs derived from induced pluripotent stem cell reprogrammed using proprietary mRNA process
  • No FDA-approved treatment for ARDS exists today
  • Preclinical activities are underway

Stem cells are an exciting new area of focus as potential therapies for ARDS, and are currently the subject of ongoing research efforts worldwide. Citius believes mesenchymal stem/stromal cells (MSCs) offer considerable promise for ARDS. Several donor-derived MSC therapies under investigation have demonstrated that MSCs may reduce inflammation, enhance clearance of pathogens and stimulate tissue repair in the lungs. MSCs have also been shown to restore endothelial and epithelial barrier integrity, enhance the clearing of fluid from the lungs, and may exhibit antimicrobial properties.

Citius is exploring the potential of its i-MSCs to overcome the limitations of MSCs derived from adult donors. Positive interim results from a proof-of-concept study demonstrate a marked improvement in i-MSC-treated animals over control animals in key clinical parameters. Learn more….