Results of Cynata’s World-First Clinical Trial of iPSC-derived CYP-001 in GvHD Accepted for Publication in Nature Medicine

15 September 2020

Release date 14 September 2020

Cynata Therapeutics Limited, a clinical-stage biotechnology company specialising in cell therapeutics, is pleased to announce that a paper describing the Phase 1 clinical trial of CYP-001 in patients with graft versus host disease (GvHD) has been accepted for publication in the prestigious Nature Medicine.1


The published trial results detail the world’s first clinical trial of an allogeneic induced pluripotent stem cell (iPSC)-derived product. CYP-001, Cynata’s lead iPSC-derived mesenchymal stem cell (MSC, also known as mesenchymal stromal cell) product candidate, broke ground by being the first MSC therapy to be produced at scale without the limitation of multiple donors through Cynata’s novel CymerusTM technology.

In the clinical trial, 15 patients with steroid-resistance acute GvHD received two infusions each of Cymerus MSCs. The trial was conducted at seven clinical centres in the UK and Australia. Publication in Nature Medicine is recognition of the importance of the findings from this study and the unique nature of Cynata’s proprietary Cymerus technology.

Key Highlights

As previously announced, key results of the clinical trial were as follows:

  • Overall Response rate by Day 100 was 87% (13/15 patients showed an improvement in GvHD severity by at least one grade compared to baseline)
  • Complete Response rate by Day 100 was 53% (GvHD signs and symptoms completely resolved in 8/15 patients)
  • Overall survival at Day 100 was 87%
  • No treatment-related serious adverse events or safety concerns were identified

The co-corresponding authors of the paper are Professor John Rasko AO (Royal Prince Alfred Hospital, Sydney) and Professor Adrian Bloor (The Christie Hospital, Manchester).

Professor Rasko commented: “iPSC-based technology facilitates the large-scale production of potential cell-based therapies in a robust and consistent manner. iPSCs have the potential to give rise to any cell in the human body. This represents the first-ever published report of safety and efficacy in a completed human clinical trial using iPSC-derived cells in any disease, worldwide. The acceptance of this manuscript by such a prestigious and high-impact journal underscores the importance of this trial to the field of cell- based medicine.”

Professor Bloor said: “Steroid-resistant GvHD is one of the most serious complications of bone marrow and blood stem cell transplantation. There is a major unmet need for an effective treatment and we are optimistic that CYP-001 can play an important role in the management of this life-threatening condition.”

Dr Kilian Kelly, Cynata’s Chief Operating Officer, said: “We are delighted that this paper has been published in Nature Medicine, which is a major endorsement of our technology and the significance of this trial. We are very grateful to the many people who contributed to this trial, including the research teams, the participating patients and their families. Further development of CYP-001 for GvHD is continuing through our global licensee for this indication, Fujifilm. We also look forward to commencing clinical trials of Cymerus MSCs in a number of other indications, including osteoarthritis and COVID-19, in the near future.”

The details of the paper, which has been published online, are as follows:
Bloor AJC, Patel A, Griffin JE, Gilleece MH, Radia R, Yeung DT, Drier D, Larson LS, Uenishi GI, Hei D, Kelly K, Slukvin I and Rasko JEJ. Production, safety and efficacy of iPSC-derived mesenchymal stromal cells in acute steroid-resistant graft versus host disease: a Phase I, multi-centre, open label, dose-escalation study.

Read the full media release.


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