[The Dong-A Ilbo] [Innovative Startup in Hongneung] StemExOne: “Ushering in a New Era of Cell Therapy with Stem Cells and Exosomes”

Stem cells are the foundational cells that give rise to the organs, bones, tissues, and blood in our bodies. When an organ or tissue is damaged, stem cells can migrate toward the injured site and contribute to the healing process. This phenomenon is known as the “homing effect.”

When stem cells exert this homing effect, they release various bioactive substances. These substances promote cellular regeneration and recovery, enhance intrinsic cellular functions, and strengthen resistance to inflammation. The beneficial effects produced by substances secreted from stem cells are collectively referred to as the “paracrine effect.” In other words, many of the therapeutic benefits associated with stem cells are believed to arise from this paracrine activity. Among the key substances involved are exosomes.

Because stem cells have a limited lifespan and are relatively difficult to handle, both laboratory research and transplantation into patients can present challenges. This raises an important question: what if, instead of transplanting the stem cells themselves, only the exosomes responsible for much of their therapeutic activity were isolated and used?

Exosomes are biological materials rather than living cells, making them easier to handle and potentially more stable. By harnessing their biological properties and therapeutic functions, researchers hope to stimulate cellular regeneration and recovery throughout the body and develop new treatments for a wide range of diseases.

One scientist who has spent more than 20 years studying stem cells and exosomes is working to turn this concept into reality. He is Ssang-Goo Cho, CEO of StemExOne, a startup based in the Hongneung Innopolis research cluster.

Cho is also a professor in the Department of Stem Cell and Regenerative Biotechnology at Konkuk University and formerly served as Director of the KU Institute of Science and Technology. He served as President of the Korean Society for Stem Cell Research in 2022 and later as its Chairman.

Cho began studying stem cells in 2003. In 2006, he encountered the landmark research on induced pluripotent stem cells by Japanese scientist Shinya Yamanaka, who later received the 2012 Nobel Prize in Physiology or Medicine for reprogramming adult somatic cells into induced pluripotent stem cells.

In 2015, a case was reported in the United States involving the use of stem cell-derived exosomes in treating chronic arthritis in a professional football player.

By 2022, interest in the field had grown substantially. Altos Labs, a company focused on cellular rejuvenation and reprogramming, raised approximately USD 3 billion in funding. Alongside stem cell and exosome-based therapeutics, the company has pursued reprogramming technologies aimed at restoring aged cells to a more youthful state.

As the therapeutic potential and commercial prospects of stem cell-derived exosomes became increasingly clear, Cho decided to draw on his more than two decades of research experience to establish StemExOne.

StemExOne’s core strengths lie in advanced stem cell culture technology and the ability to isolate exosomes with enhanced therapeutic potential from well-cultured stem cells. The company is also developing technologies to apply these exosomes as treatments for a range of diseases.

Cho’s first major objective is to develop exosome-based therapeutics for various age-related diseases.

One of the first areas he identified was the bladder. Although the bladder plays an important role in human health, it has received relatively less research attention than major organs such as the lungs or heart. As a result, conditions such as bladder pain syndrome still lack fundamentally effective treatments.

Because bladder disease can significantly affect quality of life while being less immediately life-threatening than disorders of certain major organs, Cho believes it may offer an appropriate field for testing next-generation therapies such as exosome-based treatments.

Another major area of interest for StemExOne is the brain.

The brain has traditionally been considered difficult to treat with conventional drugs because the blood-brain barrier (BBB) blocks the penetration of most therapeutic substances. Exosomes, however, are naturally produced biological vesicles and may be capable of crossing this barrier.

StemExOne is therefore conducting research on the use of exosomes to reduce inflammation in the brain, with the long-term goal of contributing to treatments for a broad range of neurological diseases.

Cho and his research team are also working to engineer exosomes into drug delivery vehicles.

StemExOne aims to load exosomes with therapeutic agents such as drugs, DNA, or mRNA to create advanced gene and drug delivery systems. In this approach, exosomes could provide not only their own biological effects but also their natural ability to travel through the body, potentially improving the delivery of therapeutic payloads to target tissues.

StemExOne is supported by a broad academic and clinical network.

Konkuk University’s Department of Stem Cell and Regenerative Biotechnology is one of Korea’s specialized academic departments dedicated to stem cell research. Researchers and students from the department, along with teams at Konkuk University Medical Center and Konkuk University Veterinary Medical Teaching Hospital, support StemExOne’s research activities.

Several physicians, including Prof. Aram Kim of Konkuk University’s Department of Urology, are involved in collaborative stem cell research with the company. Other major partners include Gangnam Severance Hospital, CHAUM, and Kyung Hee University College of Medicine.

The Hongneung Innopolis research cluster has also provided active support to StemExOne and CEO Ssang-Goo Cho.

The organization has helped facilitate joint research with Korea University Medical Center and Kyung Hee University Medical Center, while also providing training and support related to preclinical and clinical studies. In addition, it has connected StemExOne with partner institutions including the Ministry of Health and Welfare and the Korea Institute of Science and Technology (KIST).

Together with its partners, StemExOne is steadily working to turn the potential of stem cells and exosomes into real-world medical applications. However, significant challenges remain, with regulatory approval representing one of the biggest barriers.

Developing exosome-based therapeutics requires extensive cell research, animal studies, and ultimately clinical trials. Compared with stem cells, which are living cells and therefore present a wider range of safety variables, exosomes are biological materials with potentially fewer safety concerns. StemExOne had hoped to validate exosome safety and move rapidly into clinical development. However, the company says obtaining approval has been difficult because there are still few precedents for exosome-based therapies in Korea.

StemExOne has also encountered limitations under Korea’s Advanced Regenerative Bio Act, enacted in 2019. At the time, the regulatory framework primarily focused on research involving living cells. Because exosomes are not cells, they did not clearly fall within the scope of the review system. With exosome therapeutics lacking a well-defined regulatory pathway, calls from industry for regulatory reform have continued to grow, although meaningful changes are expected to take time.

Japan, by contrast, has established a regenerative medicine framework that supports research involving therapies that have not yet received full approval. Under certain conditions, physicians may administer such therapies when they determine that appropriate safety requirements have been met. This environment has enabled Japanese researchers to actively study stem cells, immune cells, and exosomes for therapeutic applications. It is also one reason some Korean patients travel to Japan for stem cell-based treatments.

Research on stem cells and exosomes is also advancing rapidly in the United States. At the time of the report, a U.S. pharmaceutical company was conducting a Phase 3 clinical trial of an exosome-based therapy for severe respiratory conditions associated with lung damage.

CEO Ssang-Goo Cho emphasizes that Korean stem cell researchers possess capabilities and expertise comparable to those of leading researchers overseas. He notes that Korean scientists are quick to adopt and apply new technologies and consistently produce high-quality research outcomes. Nevertheless, he warns that regulatory barriers could cause Korea to fall behind broader global developments in the exosome market. He argues that pathways for more active research and clinical use of stem cells and exosomes should be opened as soon as possible.

To address these challenges, StemExOne is preparing clinical development of an exosome-based therapy for interstitial cystitis, also known as bladder pain syndrome. After completing animal studies and data collection, the company planned to submit an Investigational New Drug (IND) application in 2024.

At the same time, StemExOne is pursuing applications of exosomes in skin disease treatments and cosmetic ingredients. The company is already producing exosomes and views these areas as opportunities for faster practical application.

After further validating the efficacy and safety of its exosome technologies, StemExOne plans to expand research from age-related conditions affecting the bladder to therapeutic applications involving major organs such as the brain, lungs, and liver. The company is also exploring exosome-based approaches for conditions in areas such as the salivary glands and lacrimal glands, where effective treatment options have historically been limited.

“StemExOne has already established a robust exosome production platform,” Cho said. “Our next step is to create the conditions needed to bring these technologies closer to patients and demonstrate the broader value of exosomes. Building on these achievements, we aim to lead exosome research and new drug development in Korea while successfully expanding into global markets.”

Cha Joo-kyung, IT Reporter, Donga.com
racingcar@itdonga.com