The company’s novel stem cell manufacturing process known as Cymerus has been successfully validated at Waisman Biomanufacturing’s Madison, Wisconsin-based facility after trials confirmed the stem cell manufacturing process is capable of producing MSCs for therapeutic application, consistently, efficiently and economically in a GMP production environment.
Cynata claims that the Cymerus process uses an effectively limitless starting material – a bank of induced pluripotent stem cells (iPSCs) – and a patent-protected process to derive MSCs for commercial use, which differentiates the company’s process from all existing methods of MSC production, which usually require a continuous supply of new tissue donations.
One of the major benefits of this manufacturing process is that Cynata expects to be able to produce all of its MSCs from a single iPSC bank, derived from a single blood donation. Therefore there will be no need to repeatedly source, screen, and test new donors and issues with donor-to-donor variability will not arise.
Cynata CEO Dr Ross Macdonald said: “An equity research report compiled last year by respected biotech analyst Stuart Roberts saliently noted that should Cynata demonstrate an ability to make cells at industrial scale under GMP then it would be in a position to be a ‘genuine Stem Cell Revolutionary.’”
In addition, the use of an essentially limitless starting material means that it will not be necessary for Cynata to excessively expand MSCs in culture in order to generate the vast numbers of cells required to provide commercially-viable treatments for major diseases.
Macdonald said that one of the biggest issues facing regenerative medicine companies is how to produce enough stem cells consistently and economically for clinical and commercial benefit.
“Our international manufacturing partner has now confirmed our proprietary process can achieve this in a GMP manufacturing environment. This is a key requirement for pharmaceutical companies as they move to capture the opportunities presented by stem cell medicine. An abundance of stem cells clears a path toward low cost, cutting edge cell therapy,” he added.
Cynata will now move to manufacture its GMP-grade Cymerus MSC product and expedite its clinical trial and collaboration programs. A Phase I human clinical trial of the Cymerus stem cell technology is currently in planning stage, with discussions underway with regulatory authorities to ascertain and clarify the likely regulatory path. The proposed clinical study is intended to examine the impact of these manufactured cells on patients affected by graft-versus-host disease (GvHD).
The Cynata announcement comes as the European Commission has approved the world's first stem cell derived drug. Italy-based Chiesi's Holoclar (an ex-vivo expanded autologous human corneal epithelial cells containing stem cells) has been approved for moderate to severe limbal stem cell deficiency (LSCD) due to physical or chemical burns to the eye in adults.