Cellusion Inc. announced that the first patient has received a transplanted induced pluripotent stem (iPS)-derived corneal endothelial (CE) cell substitute in its first-in-human (FIH) study of patients with regrafted cases of bullous keratopathy.
Give me a quick rundown on Cellusion.
Based in Tokyo, Japan, Cellusion was founded in 2015 as a regenerative medicine startup company with the principal goal of solving the global shortage of corneal transplant donors.
The company has developed a novel mass culture system for corneal endothelial (CE) cells produced from iPS cells as part of its mission to deliver novel therapies with cellular technology.
Of note, Cellusion has partnered with New Jersey-based Minaris Regenerative Medicine, a global contract and development manufacturing organization, after announcing a letter of intent (LOI) in October 2022.
Minaris will perform process optimization as part of the LOI to meet requirements for the FDA. If approved, they will manufacture CLS001 as a corneal endothelial cell regenerative therapy in the United States.
Tell me more about this system.
iPS-derived CE cellular therapy (CLS001) is designed as a solution for long-standing corneal transplantation problems by injecting CE cells manufactured from iPS cells.
The injection is administered via a syringe and requires a patient to remain face-down for 3 hours post-treatment.
And the trial?
The exploratory, single-arm, first-in-human clinical study (jRCTa031210199) is examining the safety and efficacy of CLS001 transplanted in three patients (ages 45-85) with regrafted bullous keratopathy. Inclusion criteria included a central CE cell density of > 500 cells/mm2 measured by specular or confocal microscope, or undetectable.
See here for the exclusion criteria.
Primary outcomes include systemic (all adverse events [AEs]) and ophthalmologic (increased IOP, unintended cell proliferation, infectious disease, cataract progression, etc.) safety endpoints, and secondary outcomes include efficacy endpoints (visual acuity [VA] and corneal thickness).
How about this transplant?
According to research collaborator Shigeto Shimmura, MD, PhD, there were no AEs observed for 3 months following transplantation of CLS001 (as of January 2023). Additionally, VA, central corneal thickness, and minimal corneal thickness has improved so far.
What’s next?
Cellusion stated it will accelerate the preparation of the sponsored clinical trial with global studies in the US and European Union to follow.