Cellusion Announces FDA Orphan Drug Designation for iPSC-Derived Therapy CLS001 Targeting Bullous Keratopathy

Recently, Cellusion, a rising star in Japan's cell therapy and regenerative medicine field, announced its ongoing research"iPSC-Derived Corneal Endothelial Cell Replacement Therapy (CLS001)" Granted Orphan Drug Designation (ODD) by U.S. FDA, for the treatment of bullous keratopathy. This designation will further accelerate the global development of CLS001.

Cellusion was born in 2015 at the Department of Ophthalmology of Keio University School of Medicine with the aim of creating innovative regenerative medicine pipelines using world-leading iPSC technology to address unmet medical needs centered around patients. To date, Cellusion has raised a total of 4.5 billion yen (33 million USD).

Cellusion is advancing the development of corneal endothelial cell substitutes led by iPSC cells, efficiently producing CLS001 from iPSC cells based on its proprietary patented technology to address the challenges of corneal endothelial transplantation. It is reported that 13 million patients worldwide are waiting for corneal transplant surgery. Notably,Cellusion signed a licensing agreement with XingSaiRuiZhen, a company under Fosun Pharma's Fosun Healthcare Capital New Drug Innovation Fund, in September 2022. XingSaiRuiZhen has obtained the rights for clinical development and future commercialization of CLS001 (CRG-101) in Greater China.。

CLS001 is an allogeneic, off-the-shelf cell therapy product. Based on iPSC-derived expansion and differentiation technology, a single production run can yield enough product for thousands of patients.Whether in terms of product quality control or production cost, it is significantly superior to similar cell therapy products on the market. It is expected to greatly alleviate the global shortage of corneal endothelial cell donors.

CLS-001 is Cellusion's proprietary technology, producing corneal endothelial cell replacement cells through the efficient differentiation of iPSC cells. iPSC cells also possess the potential to differentiate into various types of cells. Cellusion combines “CECSi Cells made from iPSC cells with excellent proliferation characteristics” and “a simple cell injection administration procedure that does not require extensive specialized training.”

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