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ERNA-201 (Rheumatoid Arthritis)

Achieved preclinical proof of concept. Further development intended through strategic partnerships.

Rheumatoid arthritis is a common and debilitating autoimmune disease that affects roughly 18 million people worldwide, 70% of whom are women. While current treatments can manage symptoms for many, a substantial number of patients - especially those with difficult-to-treat rheumatoid arthritis - continue to suffer from chronic inflammation and joint damage.

Cell-based therapies, including mesenchymal stromal cells (MSCs), have shown early promise in reducing inflammation and improving symptoms in both preclinical and early clinical studies. However, the efficacy of MSCs has been limited due to challenges with variability, manufacturing complexity, and suboptimal targeting of the inflammation site.

Ernexa Therapeutics’ candidate, ERNA-201, overcomes these challenges by leveraging synthetic MSCs derived from induced pluripotent stem cells (iPSCs). This engineered approach delivers the potent anti-inflammatory cytokine IL-10 directly to the site of inflammation, with superior consistency, enhanced functionality, and more precise targeting.

By resetting the inflammatory environment of the joints, ERNA-201 has the potential to provide more potent, lasting disease control for patients who need it most.

References

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  2. Li, Y.J. and Z. Chen, Cell-based therapies for rheumatoid arthritis: opportunities and challenges. Ther Adv Musculoskelet Dis, 2022. 14: p. 1759720X21100294.
  3. Rai, V., et al., Futuristic Novel Therapeutic Approaches in the Treatment of Rheumatoid Arthritis. Cureus, 2023. 15(11): p. e49738.
  4. He, N., et al., Mesenchymal stem cell-derived extracellular vesicles targeting irradiated intestine exert therapeutic effects. Theranostics, 2024. 14(14): p. 5492-5511.
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  6. Stoppelenburg, A.J., et al., Design of TOLERANT: phase I/II safety assessment of intranodal administration of HSP70/mB29a self-peptide antigen-loaded autologous tolerogenic dendritic cells in patients with rheumatoid arthritis. BMJ Open, 2024. 14(9): p. e078231.
  7. Nam, Y., et al., Intraperitoneal infusion of mesenchymal stem cell attenuates severity of collagen antibody induced arthritis. PLoS One, 2018. 13(6): p. e0198740.
  8. Shu, J., et al., Transplantation of human amnion mesenchymal cells attenuates the disease development in rats with collagen-induced arthritis. Clin Exp Rheumatol, 2015. 33(4): p. 484-90.
  9. Li, X., et al., Human Umbilical Mesenchymal Stem Cells Display Therapeutic Potential in Rheumatoid Arthritis by Regulating Interactions Between Immunity and Gut Microbiota via the Aryl Hydrocarbon Receptor. Front Cell Dev Biol, 2020. 8: p. 131.
  10. Abdelmawgoud, H. and A. Saleh, Anti-inflammatory and antioxidant effects of mesenchymal and hematopoietic stem cells in a rheumatoid arthritis rat model. Adv Clin Exp Med, 2018. 27(7): p. 873-880.
  11. Jung, N., et al., LC-MS/MS-based serum proteomics reveals a distinctive signature in a rheumatoid arthritis mouse model after treatment with mesenchymal stem cells. PLoS One, 2022. 17(11): p. e0277218.
  12. El-Gendy, H., et al., Comparative study between human mesenchymal stem cells and etanercept as immunomodulatory agents in rat model of rheumatoid arthritis. Immunol Res, 2020. 68(5): p. 255-268.
  13. Yang, Y., et al., Serum INF-gamma levels predict the therapeutic effect of mesenchymal stem cell transplantation in active rheumatoid arthritis. J Transl Med, 2018. 16(1): p. 165.
  14. Wang, L., et al., Efficacy and Safety of Umbilical Cord Mesenchymal Stem Cell Therapy for Rheumatoid Arthritis Patients: A Prospective Phase I/II Study. Drug Des Devel Ther, 2019. 13: p. 4331-4340.
  15. Alvaro-Gracia, J.M., et al., Intravenous administration of expanded allogeneic adipose-derived mesenchymal stem cells in refractory rheumatoid arthritis (Cx611): results of a multicentre, dose escalation, randomised, single-blind, placebo-controlled phase Ib/IIa clinical trial. Ann Rheum Dis, 2017. 76(1): p. 196-202.