Publication demonstrates the cell therapy product for Parkinson’s Disease overcomes most complex challenges in bioproduction, while demonstrating full behavioral recovery in pre-clinical model
BORDEAUX, France--(BUSINESS WIRE)--#Bioproduction--TreeFrog Therapeutics, a regenerative medicine biotech focused on using its proprietary GMP compliant technology platform, C-Stem™, to develop life-saving cell therapies, today published an article in Neurotherapeutics, demonstrating the first successful bioproduction of a Parkinson’s Disease cell therapy in a scalable bioreactor, leading to full behavioral recovery 16 weeks after transplantation using a cryopreserved 3D microtissue format.
The potential of cell therapy for Parkinson’s Disease is well documented – dating back to pioneering studies in the 1980s using fetal cell transplants – and scientific progress over the last decades provides great hope for the future. The emergence of induced pluripotent cells (iPSCs) opened new pathways and solved one of the first major challenges, cell sourcing. However, multiple hurdles remain, particularly around industrialization and no cell therapy for Parkinson’s Disease has advanced beyond clinical stage to date.
“Today’s publication demonstrates how TreeFrog Therapeutics has overcome the most complex challenges of developing a successful treatment for Parkinson’s disease using our C-Stem™ platform technology and producing a therapy containing mature dopaminergic neurons with a unique 3D format that promotes cell survival post-graft with proven pre-clinical results. We are excited to present the paper to the scientific community and look forward to many discussions on our scientific and bioproduction progress. I am proud of all the work done by Nicolas Prudon, lead author, alongside colleagues at TreeFrog”. Jens Schroeder, Chief Medical Officer, TreeFrog Therapeutics.
The paper published online today reinforces the potential of TreeFrog’s cell therapy to overcome several complex challenges, one of which being the limitation of the format. Most other existing investigational cell therapies use single-cell suspensions which have an elevated risk of inducing cell death through anoikis affecting survival and/or potency of the product post-transplantation1. The results achieved with the unique 3D format of TreeFrog’s cell therapy product demonstrate the potential to circumvent this limitation, providing protection to the more mature, sensitive cells that are neurons.
Another challenge facing companies is the bioproduction strategy. While currently only scale-out methods have been employed, which increase the potential of variability due to the manually intensive methods required, C-Stem™ production use a scale-up strategy with a stirred-tank bioreactor enhancing productivity and reducing costs. The system is commercial scale ready for the Parkinson’s disease cell therapy, with doses for hundreds of patients in one 10L bioreactor.
Finally, as in downstream processing, cryopreservation has proven to be a major challenge in the cell therapy space with fresh product performing better, while cryopreserved product resulted in a delayed time-to-effect.2 The results published today, demonstrate the same efficacy with the cryopreserved product, as the fresh product, with no time-to-effect delay.
Detailed analyses and discussion of the bioproduction process, product characterization and pre-clinical efficacy results can be found in the full paper, available here.
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About Parkinson’s Disease
Parkinson’s Disease is a progressive neurological disease affecting more than 10 million people worldwide3 with prevalence doubling in the last 25 years.4 The complex disease is the result of the degeneration of specific dopamine-producing neurons in the brain. There is a huge unmet need as current treatments provide symptomatic relief only and medication fails to control symptoms for nearly 75% of people.5 Regenerative medicine and in particular, cell therapy, holds great potential as it can replace these lost neurons, ultimately restoring function.
About C-Stem
The proprietary biomimetic C-Stem™ technology platform is based on a decade of research bringing together stem cell biology, biophysics and bioproduction. It is the world’s first GMP compliant cell encapsulation device capable of generating over 1,000 capsules per second, enabling the seeding of bioreactors up to 10 liters and delivering 15 billion iPSC cells in a single batch.
Blending microfluidics and stem cell biology, the C-Stem™ technology generates alginate capsules seeded with induced pluripotent stem cells (iPSCs) at very high speed. Engineered to mimic the in vivo stem cell niche, the capsules allow iPSCs to grow exponentially in 3D, and to differentiate into ready-to-transplant functional microtissues. The alginate is both porous and highly resistant, allowing the encapsulated iPSCs to expand and differentiate in large-scale bioreactors without suffering from impeller-induced shear stress. The breakthrough technology addresses critical challenges in the industry of scale and quality, in addition to the efficacy and safety of future cell therapy products.
About TreeFrog Therapeutics
TreeFrog Therapeutics is a French-based biotech company set to unlock access to cell therapies for millions of patients. TreeFrog is unique in its approach to cell therapy development bringing together biophysicists, cell biologists and bioproduction engineers to address the challenges of the industry — producing and differentiating cells of quality at unprecedented scale, cost-effectively. To realize their mission of Cell Therapy for all, TreeFrog operates a business model that includes its own therapeutic programs and partnerships with leading biotech and industry players in other areas. In the last 3 years, the company has raised $82 million to advance a pipeline of stem cell-based therapies in regenerative medicine. In 2022, the company opened technological hubs in Boston, USA, and Kobe, Japan, to drive the adoption of the C-StemTM platform and establish strategic alliances with leading academic, biotech and industry players.
1 Gilmore A.P., Anoikis, Cell Death Differ, 12 (2005), pp. 1473-1477
2 Hiramatsu S., Morizane A., Kikuchi T., Doi D., Yoshida K., Takahashi J., Cryopreservation of induced pluripotent stem cell-derived dopaminergic neurospheres for clinical application, J Park Dis., 12 (2022), pp. 871-884
3 Statistics | Parkinson’s Foundation
4 Parkinson disease (who.int)
5 www.parkinsons.org.uk
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