Expedited Development Enabled by RMAT and Breakthrough Therapy Status
Clinical Trial Initiation in 2026, FDA Approval Target by 2030
LOS ANGELES, CA / ACCESS Newswire / December 23, 2025 / Nature Cell Co., Ltd., a biotechnology company focused on adult stem cell therapies (Chairman: Jeong-Chan Ra), announced the full-scale advancement of its stem cell therapy Vascostem for the treatment of Critical Limb Ischemia (CLI) patients in the United States. The company aims to accelerate clinical development through the FDA's Regenerative Medicine Advanced Therapy (RMAT) designation. Development activities in the U.S. will be led by its wholly owned subsidiary Nature Cell America, Inc.
Critical Limb Ischemia (CLI) is the end-stage of peripheral arterial disease, characterized by severe pain, tissue damage, and infection. Without adequate treatment, CLI can lead to limb amputation and can be fatal. In the United States alone, approximately 150,000 patients undergo leg amputations each year due to CLI. The number of so-called "no-option CLI patients," who do not respond to existing treatments, continues to rise, highlighting a significant unmet medical need and market opportunity.
Vascostem is an autologous adipose-derived stem cell therapy developed based on mechanisms that promote angiogenesis and ischemic tissue recovery, primarily targeting severe CLI patients with limited treatment alternatives.
In animal studies, Vascostem demonstrated a dose-dependent reduction in amputation rates. While the control group showed 100% amputation (12 out of 12 cases at above-knee level), the low-dose group showed 16.7% (2 out of 12), the medium-dose group 25% (3 out of 12), and the high-dose group 0% (0 out of 12) amputation rates.
Particularly noteworthy are the clinical trial results. In human clinical trials using the medium dose (5 million stem cells per kg body weight), not a single patient among 15 total participants required above-knee amputation at the 6-month follow-up (0/15, 0%). This demonstrates a clear correlation between the amputation prevention effects observed in animal studies and clinical outcomes. The complete prevention of Major Amputation (above the knee) holds significant clinical importance. Among the 15 patients, 5 (33.3%) underwent only Minor Amputation (toes or partial foot), representing a breakthrough clinical achievement difficult to attain with conventional treatments.
Substantial safety and efficacy data have already been generated through animal and clinical studies. Notably, for Buerger's disease indication, Vascostem has received Orphan Drug designation from the U.S. FDA, establishing a foundation for reduced development risk and market exclusivity.
Vascostem for U.S. clinical trials will be manufactured at the Biostar Stemcell Campus GMP Center located in Baltimore, Maryland. By securing GMP manufacturing infrastructure within the U.S. in 2026, the company will establish manufacturing and quality systems compliant with FDA standards while ensuring supply chain stability for the future commercialization phase. Johns Hopkins Hospital and University of Maryland Medical Center, both in close proximity to the Baltimore GMP Center, are being considered as primary candidate sites for clinical trials.
Nature Cell anticipates that Vascostem will receive FDA RMAT designation in the first half of 2026. RMAT designation provides benefits such as early consultation with the FDA and priority review, contributing to shortened development timelines and enhanced approval probability. The company has presented a specific timeline: submission of an Investigational New Drug (IND) application in the second half of 2026, followed by initiation of U.S. clinical trials within the same year. If development proceeds smoothly, the company expects to enter Phase 3 clinical trials in 2028, with FDA approval in 2030 and U.S. market launch in 2031.
Nature Cell's Chairman Jeong-Chan Ra stated, "Limb amputation due to critical limb ischemia is a grave issue that threatens not only patients' quality of life but their very lives. Building on our successful U.S. development experience with Jointstem, we are reviving Vascostem's previously suspended journey toward U.S. approval."
He continued, "Based on already secured animal study and clinical trial data, as well as our GMP manufacturing infrastructure in the U.S., we are pursuing an accelerated development strategy through RMAT designation, targeting FDA approval by 2030. Given our existing research achievements, the probability of success is very high - it's merely a matter of time to commercialization. We are committed to providing this limb-saving and life-saving stem cell therapy to American patients as quickly as possible, solidifying Nature Cell's global leadership in stem cell therapeutics."
For inquiries, contact Biostar's Marketing Department at 02-6978-9209, 02-6978-9225.
SOURCE: Nature Cell
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