Organoid Science Receives World's First IND Approval for Intestinal Organoid Therapy in Crohn's Disease
Key Insights
Organoid Science (search) received IND approval from Korea's Ministry of Food and Drug Safety (search) for ATORM-C (search), marking the world's first organoid-based regenerative therapy to enter clinical trials.
The Phase 1 trial will evaluate ATORM-C (search) in Crohn's disease (search) patients with colonic ulcers, using autologous intestinal organoids to regenerate damaged mucosal tissue.
This milestone follows an 8.3 billion won technology transfer from KRIBB (search) to Organoid Science (search), bringing years of stem cell organoid research into commercial development.
Organoid Science (search) has achieved a global first in regenerative medicine, receiving approval from Korea's Ministry of Food and Drug Safety (search) to begin Phase 1 clinical trials of ATORM-C (search), an intestinal organoid therapy for Crohn's disease (search) patients with colonic ulcers. The IND approval, announced on March 16, represents the world's first organoid-based regenerative therapy to enter the clinical trial stage.
Breakthrough Technology for Intractable Disease
ATORM-C (search) is composed primarily of intestinal organoids manufactured using three-dimensional organoid culture technology from tissue-derived stem cells extracted from patients' intestinal tissue. The therapy works by promoting regeneration of damaged intestinal mucosal tissue to treat refractory ulcers that do not respond to existing therapies, thereby reducing intestinal inflammation and preventing disease progression.
The treatment is directly transplanted into damaged intestinal mucosa (search), where it differentiates into actual intestinal epithelial cells (search) and induces regeneration of damaged tissue. This approach addresses a critical unmet medical need in Crohn's disease (search), where existing treatments focus on controlling inflammation but often leave untreated ulcers that can lead to severe complications including intestinal strictures and perforation.
Clinical Trial Design and Objectives
The Phase 1 trial will be conducted at Asan Medical Center in Seoul and will assess the tolerability and safety of ATORM-C (search) after administration. The study aims to determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D), while concurrently evaluating exploratory efficacy. Between 9 to 18 patients are expected to participate, with safety and efficacy monitored for up to 24 weeks following administration.
The IND approval came approximately three months after the application was submitted on December 30, demonstrating the regulatory pathway's efficiency for this innovative therapeutic approach.
Foundation Built on Decades of Research
This clinical milestone builds upon significant foundational research conducted by the Korea Research Institute of Bioscience and Biotechnology (search) (KRIBB (search)). The institute's research team, led by Son Miyoung, head of the Stem Cell Convergence Research Center, published manufacturing technology for mature intestinal organoids derived from human pluripotent stem cells in Nature Communications in 2018, followed by advanced tissue regeneration-based technologies in 2024.
In a landmark technology transfer deal, KRIBB (search) transferred its "source technology for human intestinal organoid-based regenerative therapeutics and drug evaluation platforms" to Organoid Science (search) for approximately 8.3 billion won, plus running royalties based on future sales. The transfer package includes three core patents and standard operating procedures for manufacturing, quality evaluation, cryopreservation, and thawing.
Addressing Treatment Gaps in Inflammatory Bowel Disease
Crohn's disease (search) represents a significant therapeutic challenge as a chronic inflammatory bowel disease (search) characterized by recurring inflammation and ulcers in the intestinal mucosa (search). While various drugs are currently available, the chronic nature of the disease means that untreated ulcers often remain in a significant number of patients, exacerbating their condition.
The KRIBB (search) research team integrated into a single platform the production of mature intestinal organoids from pluripotent stem cells at a level comparable to actual intestinal tissue, technologies that improve engraftment and regenerative efficiency after transplantation, and technologies for large-scale culture, cryopreservation, and thawing. This establishes a foundation for developing an off-the-shelf cell therapy product that can be manufactured and stored in advance and administered immediately when needed.
Commercial and Scientific Implications
"The approval of this IND is significant as it marks the first time an organoid-based regenerative therapy has entered clinical trials in Korea," stated Jongman Yoo, CEO of Organoid Science (search). "We expect that this new regenerative approach using organoids can offer new possibilities for the treatment of refractory diseases that do not adequately respond to existing therapies."
Beyond therapeutic applications, Organoid Science (search) plans to expand the technology as a New Approach Methodologies (NAMs) platform to evaluate drug efficacy and toxicity, potentially reducing animal testing in drug development and building evaluation systems that precisely reflect the human intestinal environment.
Son Miyoung emphasized the significance of this achievement: "This technology transfer marks an important starting point where KRIBB (search)'s accumulated source technologies for human intestinal organoids are combined with the commercialization capabilities of industry to be applied to real patient care. We will continue follow-up research so that this can provide tangible benefits for patients with intractable intestinal diseases."