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news.Australian stem cell and regenerative medicine firm, Cynata Therapeutics, has secured approval from the UK Medicines and Healthcare products Regulatory Agency (MHRA) to go ahead with its Phase 1 clinical trial of CYP-001 in patients with steroid-resistant graft-versus-host disease (GvHD).
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CYP-001 is Cynata's lead Cymerus mesenchymal stem cell (MSC) product.
Cynata plans to conduct the Phase 1 clinical trial, which is entitled "An Open-Label Phase 1 Study to Investigate the Safety and Efficacy of CYP-001 for the Treatment of Adults With Steroid-Resistant Acute Graft Versus Host Disease", at a number of leading clinical centres in the UK and Australia. Additional centres in other jurisdictions are also being considered.
The trial will aim to recruit approximately 16 participants who have undergone a bone marrow transplant or similar procedure, and were subsequently diagnosed with steroid-resistant Grade II-IV acute GvHD.
The Company believes that this will be the world's first clinical trial involving a therapeutic product derived from allogeneic induced pluripotent stem cells (iPSCs).
Pluripotent stem cells are the most versatile cells of all, having the ability to reproduce themselves indefinitely, and also differentiate into any other type of cell in the body. iPSCs have very similar characteristics to embryonic stem cells (ESCs), but without the ethical controversies associated with ESCs, since they are derived from adult cells rather than from embryos.
There has been enormous interest globally in the development of iPSC-derived therapies for a number of years. This trial represents a major milestone in the field of regenerative medicine. Results from the clinical trial will provide further insight into the use of Cynata's proprietary technology for the potential treatment of various diseases.
Cynata vice president of product development Kilian Kelly said: "We are delighted that the MHRA has approved our clinical trial. Not only does this enable us to start providing our highly promising therapy to patients with a particularly devastating disease, it also provides clear validation of our manufacturing process and preclinical development program, from one of the most highly regarded regulatory authorities worldwide."
Cynata CEO and managing director Ross Macdonald said: "The MHRA approval, combined with Cynata's developing business relationships with FUJIFILM and with apceth, cements the Company's commercial and technical leadership in second generation MSC technologies and it sets Cynata on a path to sustainable success."