The number of domestic corporations venturing into organoids (mini-organs) is increasing. They are evolving from alternatives to animal testing into treatments that restore damaged areas due to diseases. The scope of organoid usage is expanding from research to therapy. In line with this, regulatory authorities are also starting to revise related systems, indicating that an 'organoid industry ecosystem' is being created in the country.
According to the industry on the 1st, domestic bio corporations such as Samsung Biologics, Organoid Science, Kangstem Biotech, ROKIT Healthcare, CellInCells, and Corestemchemon have successively entered the organoid market.
Organoids mean organ (organ) analogs (oid) and are also known as mini-organs because stem cells that grow into all human body cells are cultivated into three-dimensional structures similar to organs. They are attracting attention as a technology that better reflects real organs than human cells cultivated in two dimensions, providing an alternative to animal testing.
According to market research institution The Insight Partners, the global organoid market is expected to grow from approximately $2.8 billion (about 3.9 trillion won) in 2023 to $10 billion (about 13.9 trillion won) by 2030, with an average annual growth rate of around 22%.
◇The U.S. FDA allows replacement of animal testing with organoids
Currently, the organoid market can be broadly divided into three areas: research and clinical trials for developing new drugs and toxicity evaluation, utilization for patient transplantation as cell and tissue regeneration therapies, and services for producing and cultivating organoids along with other applications.
Samsung Biologics has launched the 'Samsung Organoid' service that tests new drug candidates using organoids as it enters the contract research organization (CRO) business. Until now, it focused on contract development and manufacturing (CDMO) of biopharmaceuticals for customers, but it is expanding its business scope to include clinical trials.
The U.S. Food and Drug Administration (FDA) is also noted as a background for the launch of Samsung Organoid, as it decided to gradually abolish the requirement for animal testing at the first stage of clinical trials earlier this year. Organoids have the advantage of taking less time than animal testing and being free from ethical issues, while being able to more closely mimic the human body environment.
Samsung Biologics plans to first assist in finding anti-cancer new drug candidates using cancer organoids. The new drug candidates are injected into organoids made by cultivating cancer patient cells to evaluate efficacy.
Organoids are expected to not only replace animal testing but also significantly take on human clinical trials. This is because technologies such as consolidation for creating blood vessels in organoids and connecting multiple organoids have been developed.
Until now, organoids have closely resembled the form and structure of organs but have had limitations in that they lack their own blood vessels, which prevents them from perfectly replicating human metabolism. Once organoids are equipped with blood vessels, they can show not only cellular responses but also changes in organ function.
The same applies to consolidations. To test drugs for treating kidney diseases, both kidney organoids and liver organoids are needed. Since drugs are processed in the liver, having liver organoids is essential to understand both efficacy and side effects. Additionally, heart organoids are needed to circulate blood.
◇Evolving from clinical trial tools to therapies
Some domestic corporations are also challenging the development of organoid regenerative therapies. These therapies involve transplanting organoids made from patient cells into organs or tissues damaged by disease to assist in regeneration, which differs from traditional stem cell therapies that require differentiation into the corresponding tissue or cells. Organoids can directly replace the damaged area.
Organoid Science is developing treatments for intractable diseases based on organoid models for organs and diseases such as the intestine, salivary glands, liver, and endometrium. The organoid being developed by the company, ATORM-C, is an intestinal organoid. It involves harvesting normal intestinal mucosal tissue from a patient via endoscopy, cultivating the organoid, and injecting it into damaged tissue. The goal is to develop it as a treatment for proctitis and inflammatory bowel disease.
CellInCells is also in the process of developing organoid regenerative therapies for skin and cartilage regeneration and angiogenesis. One of these is the candidate substance for treating atrophic scars, 'TRTP-101'. It received approval from the Korean Ministry of Food and Drug Safety for clinical phase 1 trial plans in August 2023, and the domestic phase 1 trial was completed last December. The company has applied for clinical phase 2 plans this year.
TRTP-101 promotes the secretion of extracellular matrix (ECM) and growth factors at the administration site. This leads to active production of elastin, collagen, and other components that make up the skin, facilitating skin regeneration. The company reported a 26% skin regeneration effect based on a 12-week treatment in clinical phase 1.
◇Formation of a corporate consortium, regulatory authorities also preparing systems
ROKIT Healthcare has commercialized a diabetes foot regeneration technology that produces extracellular matrix (ECM) from adipose-derived cells as a patch to be applied to wounds. About 15-25% of diabetes patients develop diabetic foot ulcers due to complications that impede blood flow and damage skin tissue on the legs. Even small injuries can worsen to the point of requiring leg amputation and can even lead to death. The goal is to treat diabetic foot ulcers by applying organoid patches to the wounds.
Kangstem Biotech is developing pancreatic organoids following skin organoids. The company is also pursuing a project to collaboratively conduct experiments such as gene analysis and efficacy assessments of undisclosed new drug substances being developed by global pharmaceutical companies, utilizing the skin organoid disease platform.
Domestic organoid development companies have also organized efforts in preparation for industrialization. Last month, the country's first organoid specialized industry-academic-research consortium, 'K-organoid Consortium', was launched. Until now, corporations, universities, and research institutes have conducted organoid research individually, but in the future, they aim to share information and resources, engage in joint research and development (R&D), and communicate with regulatory authorities. This also signifies that an industrial ecosystem is being built to connect the research achievements of domestic companies to commercialization.
Regulatory authorities are also preparing relevant systems in the country. Lee Han-gil, a professor at Ewha Womans University, noted that 'organoid therapies represent a different paradigm from existing pharmaceuticals, and relevant institutions such as the Ministry of Food and Drug Safety are working on establishing classification standards for clinical trials and research and development of organoid therapies.'