Organoid Science Receives IND Approval for Phase 1 Trial of Organoid-Based Regenerative Therapy

Autologous Adult Stem Cell-Derived Therapy ‘ATORM-C’
Phase 1 Clinical Trial Initiated for Crohn’s Disease Patients

Organoid Science announced on March 16 that the Ministry of Food and Drug Safety has approved its IND (Investigational New Drug) application for a Phase 1 clinical trial of ‘ATORM-C’, an intestinal organoid therapy derived from autologous adult stem cells, targeting patients with Crohn’s disease accompanied by colonic ulcers.

The company explained that this approval came approximately three months after the application was submitted on December 30 last year, marking the world’s first case of an organoid-based regenerative therapy entering the clinical trial stage.

Crohn’s disease, a chronic inflammatory bowel disease, is a refractory disorder characterized by recurring inflammation and ulcers in the intestinal mucosa. If left untreated, it can progress to severe complications such as intestinal strictures and perforation. Although various drugs are currently used, due to the nature of chronic disease, untreated ulcers often remain in a significant number of patients, exacerbating the condition. As a result, there is an ongoing medical demand for new therapeutic approaches.

ATORM-C is primarily composed of ‘intestinal organoids’ manufactured using three-dimensional organoid culture technology from tissue-derived stem cells extracted from the patient’s intestinal tissue. By promoting the regeneration of damaged intestinal mucosal tissue, it treats refractory ulcers that do not respond to existing therapies, thereby reducing intestinal inflammation and preventing disease progression. ATORM-C is directly transplanted into the damaged intestinal mucosa, where it differentiates into actual intestinal epithelial cells, thereby inducing regeneration of the damaged tissue.

The Phase 1 clinical trial, which will be conducted at Asan Medical Center in Seoul, will assess the tolerability and safety of ATORM-C after administration, determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D), and concurrently evaluate exploratory efficacy. A total of 9 to 18 patients are expected to participate in the trial, with safety and efficacy monitored for up to 24 weeks following administration.

Jongman Yoo, CEO of Organoid Science, stated, “The approval of this IND is significant as it marks the first time an organoid-based regenerative therapy has entered clinical trials in Korea. 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.”

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