[Edaily Reporter Kim Hyung-il ] #ENCell Co., Ltd., a company specializing in cell and gene therapies (CGT), announced on the 10th that it has registered a domestic patent for a core technology that selects stem cells with superior therapeutic efficacy.

ENCell Co., Ltd. plans to combine its proprietary cell culture platform with high-efficacy cell screening technology to enhance the quality consistency and manufacturing competitiveness of its cell therapies and expand the development of treatments for muscle disorders such as sarcopenia. Even for stem cell therapies derived from the same tissue, therapeutic efficacy can vary depending on the donor and the condition of the cells. This is why ensuring quality consistency is considered a critical challenge in the cell therapy development process.

Furthermore, ENCell Co., Ltd. identified biomarkers—biological indicators—characteristic of cells with superior therapeutic efficacy by comparing and analyzing mesenchymal stem cells (MSCs) obtained from multiple donors. This patent covers a technology that utilizes these biomarkers to pre-select stem cells with high therapeutic efficacy.

ENCell Co., Ltd. also confirmed the correlation between these biomarkers and muscle protection and regeneration effects through cell and animal experiments. Stem cells with high biomarker levels effectively inhibited the death of damaged muscle cells. In animal models of muscular dystrophy, they also demonstrated relatively higher muscle protection and tissue recovery effects.

The scope of the patent rights includes not only methods for selecting high-efficacy stem cells, the selected stem cells, and treatments for muscle diseases utilizing them, but also analytical compositions and kits.

This technology is expected to be utilized in conjunction with ENCell Co., Ltd.’s proprietary cell culture platform, “ENCT (ENCell Technology).” ENCT is a technology that has reduced the culture period—which previously took more than 40 days to produce over 1 billion stem cells—to approximately 20 days. It is characterized by minimizing cell function decline while increasing production efficiency by shortening the culture period.

Last September, ENCell Co., Ltd. received a decision granting a U.S. patent for this culture technology and secured a two-year extension of the National New Technology (NET) designation for its EN001 manufacturing technology. The company’s strategy is to integrate this high-efficacy cell screening technology with its existing culture technology to simultaneously improve production efficiency and ensure consistent quality. By pre-selecting cells with superior therapeutic effects, the company expects to reduce efficacy variability in cell therapies and strengthen its competitiveness in the development of treatments for muscle diseases.

ENCell Co., Ltd. is also pursuing the expansion of indications for “EN001,” an allogeneic mesenchymal stem cell therapy candidate currently under development. EN001 is an allogeneic mesenchymal stem cell therapy derived from umbilical cord blood and is currently undergoing a Phase 2a clinical trial in patients with Charcot-Marie-Tooth disease (CMT).

Previously, exploratory results from a pooled analysis of 18 patients who participated in three early-stage studies showed that 15 patients (83.3%) demonstrated an improvement of at least 20% in their neuropathy assessment scores. However, these are exploratory analysis results based on participants in early-stage studies.

Building on its experience in clinical development for rare diseases, ENCell Co., Ltd. is expanding its development portfolio to include muscle disorders such as sarcopenia. In March, the company received approval from the Ministry of Food and Drug Safety to amend its Phase 1 and 2 clinical trial plan for sarcopenia, and plans to evaluate the effects of EN001 on improving physical function in 114 patients during the Phase 2 trial.

Recently, the company also confirmed the potential for improvement in muscle regeneration-related indicators in animal models of aging. Based on these findings, the company is considering expanding its research into the fields of frailty and anti-aging.

An ENCell Co., Ltd. official stated, “With this patent registration, we have secured the technological foundation to objectively screen for stem cells with superior therapeutic efficacy,” adding, “By leveraging our proprietary ENCT culture platform and high-efficacy cell screening technology, we will strengthen the quality consistency and manufacturing competitiveness of our cell therapies and accelerate the development of treatments for various muscle disorders, including sarcopenia.”