Corporations that had been in a prolonged slump in cell and gene therapies (CGT) are recently accelerating commercialization and global expansion. After experiencing waves of controversy over theme stocks, canceled approvals, and poor business results, a downward trend in stock prices has continued for several years, prompting attention to whether they can create a new rebound opportunity.
Cell and gene therapy is a third-generation biopharmaceutical that fundamentally treats diseases by modifying or altering the patient's genes or cells. It is cited as the key to treating difficult and rare diseases that have been challenging to treat due to its completely different mechanisms of action from existing drugs. According to global market research firm Frost & Sullivan, the cell and gene therapy market is expected to grow more than threefold from $16.33 billion (approximately 23.8 trillion won) in 2023 to $55.59 billion (approximately 81.39 trillion won) by 2026.
◇MEDIPOST CATIS, challenges in the U.S. and Japan
MEDIPOST, considered a first-generation cell and gene therapy corporation in Korea, is seeking growth breakthroughs in the U.S. and Japan for its stem cell treatment 'CATIS.' On the 29th, MEDIPOST announced that it had invited industry stakeholders to hold a 'Train-the-Trainer Program' ahead of its entry into Phase 3 clinical trials for patients with degenerative and recurrent traumatic arthritis.
This program is an event that will invite clinical trial participants who are authorities in the U.S. orthopedic field over two sessions in April and May to educate them on CATIS surgical methods and the overall clinical trial procedures. It was held at Ewha Womans University Medical Center, one of the 10 institutions that participated in the domestic Phase 3 clinical trial.
Seung-jin Lee, Deputy Minister of MEDIPOST and co-representative of its U.S. corporation, noted, "This training program was prepared to share treatment methods before the Phase 3 trial and strengthen cooperation with U.S. medical professionals."
CATIS is the world's first allogeneic umbilical cord blood-derived stem cell treatment, which received item approval in 2012. It was developed as a treatment that can be used on other individuals from stem cells found in umbilical cord blood. As of last year, the cumulative number of patients treated exceeded 32,000.
MEDIPOST was established in June 2000 by chairman Yang Yoon-sun, a specialist from Samsung Medical Center, and was listed on the KOSDAQ in July 2005. Although the company garnered significant attention in the market early on for its technological prowess, its stock price has faced a downward trend for several years due to controversies over political theme stocks and poor performance.
The company secured funds for global clinical trials of CATIS through a capital increase in 2023. MEDIPOST stated that it is currently focusing on preparing for a clinical approval application (IND) with the U.S. Food and Drug Administration (FDA) and that it has completed administering treatment to the last patient in the Phase 3 trials in Japan.
◇KOLON InVossa 'confirms safety'… Is the U.S. market opening?
TG-C, a treatment for osteoarthritis that faced a crisis of having its domestic approval revoked due to safety controversies, is also approaching U.S. approval. KOLON TissueGene, established in 1999, announced at the International Osteoarthritis Congress (OARSI) held in Songdo, Incheon from the 24th to the 27th that no cases of cancer have been found in long-term follow-up studies lasting up to 15 years.
TG-C received domestic approval in 2017 under the name InVossa. It garnered considerable attention both domestically and internationally as the world's first gene therapy for osteoarthritis. However, it was later revealed that different cells had been mixed into clinical trials, leading to the revocation of domestic approval in 2019. This was because the cells confirmed during U.S. Phase 3 trials were kidney cells rather than the cartilage cells disclosed in the initial clinical plan.
In May of that year, the U.S. FDA suspended the clinical trial, and the South Korean Ministry of Food and Drug Safety (MFDS) revoked the item approval in July. The FDA allowed the resumption of TG-C's U.S. Phase 3 trial in 2020, accepting KOLON TissueGene's claim that radiation would block cell division and eliminate cancer risk. KOLON TissueGene stated that it plans to apply for a biologics license application (BLA) to the FDA as soon as it completes follow-up observations of Phase 3 trial patients in the second half of next year.
◇Benefitting from the Advanced Regenerative Medicine Act… Corporations rushing to obtain facility approvals
Other cell and gene therapy corporations are seizing business opportunities by quickly obtaining facility approvals after the so-called revision of the Advanced Regenerative Medicine Act (Law on the Safety and Support of Advanced Biopharmaceuticals) came into effect in February. The revision of the act eased regulations to allow patients participating in clinical trials to receive the stem cell and gene therapies currently in development.
VaxCell-Bio, listed on the KOSDAQ through a technology exception in 2020, announced on the 28th that it had received authorization from the MFDS for a facility that processes autologous bone marrow-derived cells. It also stated plans to conduct clinical trials on autologous bone marrow-derived cells in terminal heart failure patients, marking a first in Korea.
ViGenCell also obtained approval for its cell processing facility from the MFDS in February. Established in 2013 as the first technology holding company of Catholic University, ViGenCell was once incorporated into Boryung Pharmaceutical and changed its name to Boryung ViGenCell. The company is currently conducting Phase 2 and Phase 1 trials for its treatments 'VT-EBV-N' and 'VT-Tri(1)-A,' both made from immune cells, namely natural killer (NK) cells and T cells.