Cell and gene therapies that have been unable to gain traction due to repeated controversies in Korea are accelerating commercialization in the United States and Japan. Kolon TissueGene and Nature Cell are representative examples.
Kolon TissueGene noted on the 8th that it plans to start Phase 1 clinical trials for the cell and gene therapy 'TG-C' for spinal indications in the U.S. starting in the second half of this year. Following a Phase 3 clinical trial involving knee osteoarthritis patients in the U.S., it is set to expand the indications for clinical trials.
TG-C is a pharmaceutical made by mixing two components: one liquid containing human cartilage cells and a second liquid consisting of transformed cartilage cells containing a gene (TGF-β1) that helps cartilage cells grow. The company stated that it will complete the contract for selecting a clinical trial organization (CRO) by the first quarter of next year after undergoing the necessary procedures for U.S. clinical trials in the second half of this year. It plans to administer TG-C to 24 patients with spinal indications at clinical sample dosing hospitals starting in the second half of next year.
TG-C was originally approved as a cell and gene therapy for osteoarthritis under the name Inbonetics in 2017 by the Ministry of Food and Drug Safety. However, in 2019, it was revealed that the second component contained kidney cells instead of cartilage cells, leading the ministry to decide to suspend sales and revoke product approval that year.
The U.S. Food and Drug Administration (FDA) had a different assessment. After about a year of investigation, the FDA determined that the possibility of safety issues arising from the mixed cells was low and allowed the resumption of Phase 3 clinical trials in the U.S. Kolon TissueGene began Phase 3 trials targeting knee osteoarthritis patients in the U.S. in April 2020 and completed patient dosing in July of last year. Currently, it is under two years of follow-up observation, with the expected end of follow-up observation in the second half of next year.
The FDA also approved the expansion of clinical trials for spinal indications, stating that there were no safety issues with TG-C, similar to those seen in the knee trials. Lee Moon-jong, CEO of Kolon TissueGene, stated, "We were able to significantly shorten the time to enter the spinal trials based on the data from the knee clinical trials for TG-C."
Expanding indications increases the treatment range and the potential to become a global new drug. Jeon Seung-ho, CEO of Kolon TissueGene, said, "Spinal diseases are representative degenerative musculoskeletal disorders, along with knee osteoarthritis, with a very large number of patients and currently limited treatment options," adding, "The initial clinical trial results indicate promising technology transfer opportunities."
Nature Cell announced that its mesenchymal stem cell therapy 'Jointstem' received designation as an 'innovative therapy' from the U.S. FDA last April. The innovative therapy designation is a system that prioritizes the review process for market authorization to accelerate the development of therapies. The company stated that this designation was granted based on the results of a Phase 3 clinical trial and follow-up observations over three years involving severe knee osteoarthritis patients.
According to the company, Jointstem, made from stem cells extracted from the patient's fat, is administered once into the knee joint cavity to promote cartilage regeneration in patients with severe knee osteoarthritis. The company explained that this can sustain effects of pain reduction and joint function improvement for at least three years. Last October, it also received designation as a 'regenerative medicine advanced therapy (RMAT)' from the FDA.
Jointstem has not gained recognition in Korea due to ongoing controversies. Previously, the company had applied for conditional product approval for Jointstem as a treatment for degenerative arthritis to the Ministry of Food and Drug Safety in June 2017, but this was rejected the following year due to a determination of insufficient efficacy. In 2021, a formal product approval was requested but was again rejected in 2023.
During this time, company executives were also indicted for allegedly manipulating stock prices to gain undue profits during the conditional product approval application process for Jointstem. However, all defendants, including Nature Cell Chairman Ra Jeong-chan, were acquitted by the trial court in 2020, the appellate court in 2022, and the Supreme Court in 2023.
Nature Cell is targeting the Japanese market. Since 2015, Japan has legally allowed stem cell therapy to be performed unrestricted in medical institutions. Jointstem is used for 16 diseases in Japan, ranging from arthritis to Parkinson's disease. Nature Cell participated in the 2025 World Expo held in Osaka, Japan, last April, operating an independent exhibition booth together with its Japanese affiliate Japan Angel Stem Cell.
Additionally, SillaJen's anti-cancer virus treatment candidate 'Pexa-Vec' is seeking a comeback after experiencing a global Phase 3 failure for liver cancer patients in 2019, changing its target to other cancer types, and moving forward with a collaboration with Regeneron in the U.S. Pexa-Vec has modified the vaccinia virus to only infect cancer cells.
Last year, SillaJen announced that its clinical trial results combining Pexa-Vec with Regeneron's immuno-oncology drug Libtayo (component name Semiplimab) showed superior therapeutic effects compared to Libtayo monotherapy in kidney cancer patients. The company stated that it is seeking options for transferring the technology of Pexa-Vec overseas.