SAN FRANCISCO, Calif. — By Kim Chan-hyuk/Korea Biomedical Review correspondent — ENCell is expanding its cell and gene therapy (CGT) contract development and manufacturing (CDMO) business based on its experience in producing CAR-T cell therapies with global pharmaceutical companies while accelerating the technology transfer of its mesenchymal stem cell (MSC) therapy, EN001.
“Building a global network of CGT CDMOs and discussing the technology transfer of our pipeline EN001 were the two reasons for attending the JPM 2025 conference,” said Oh Choong-seob, director of strategic planning at ENCell, at the 43rd J.P. Morgan Healthcare Conference on Tuesday (local time).
After over a decade of experience in drug development and technology evaluation at Celltrion, where he helped build a portfolio of next-generation drugs, Oh joined ENCell in 2023 to lead pipeline value maximization and new business development.
“Technology exchange among CDMOs is important because the regulatory agency in each region has different requirements,” Oh said, explaining the importance of building a global network of CGT CDMOs. “We can collaborate and share technical know-how when conducting clinical trials in different regions.
Oh added that this event will help ENCell build a network with CDMOs in Japan, the United States, and other countries.
ENCell also strives to strengthen its CDMO competitiveness by, for instance, pushing for collaboration with Sartorius Korea on the development of the AAV production process.
“When a client comes back to us again after working with us once, it means that trust has been built,” Oh said, noting that global pharmaceutical companies, including Novartis and Janssen, have placed orders for semi-finished CAR-T cell therapy products.
“Our experience working with global pharmaceutical companies helps us build new partnerships. For example, it is easier for other pharmaceutical companies to make decisions when they have a reference that we have produced half-finished Kymriah treatments without any problems,” he added.
Commenting on the recent state of the cell and gene therapy CDMO industry, Oh said, “It is true that CGT CDMOs boomed during Covid-19 due to the production of vaccines and have since slowed down somewhat. However, CGT is not just a trend, but the direction the industry must go, so it is bound to grow in the long run.”
He also shared a memorable moment from a session at this year’s JPMorgan Healthcare Conference, saying, “A founder of a large venture fund said, ‘The CGT space is looking out the window right now, and it looks like the window is in the basement,’ which resonated with the audience.”
Oh emphasized that regardless of the short-term business environment, ENCell will continue to focus on process development and securing technology.
Oh expressed excitement about the “Act on Safety and Support for Advanced Regenerative Medicine and Advanced Biopharmaceuticals,” which will be implemented this year.
“We plan to make the most of the new markets that the new law will open up,” he said. “We are looking to maximize synergies through our network with CDMOs. We had discussions at the J.P. Morgan Healthcare Conference to flesh out our plans.”
‘FDA’s recent nod provides a green light for EN001’s challenge as the first CMT disease drug’
Regarding technology transfer discussions for EN001, a mesenchymal stem cell therapy, Oh said, “We have the most clinical data in the CMT (Charcot-Marie-Tooth disease) indication, and we are focusing on this part of the meeting. We are also discussing with partners the possibility of expanding to other indications, such as DMD (Duchenne muscular dystrophy) and sarcopenia.”
“Japanese companies are interested in stem cell therapies, and U.S. and European companies with experience in rare diseases are interested,” Oh said. In June, ENCell will attend the Bio International Convention (BioUSA) in Boston, Mass., and a partnering event organized by the Alliance for Regenerative Medicine (ARM) in October.
Last December, Oh presented the results of the EN001 phase 1a clinical trial in patients with CMT disease type 1A (CMT1A) and DMD at the 2024 Joint International Congress of the Korean Society of Medical Genetics and Genomics (KSMGG) and the East Asian Union of Human Genetics Societies (EAUHGS).
“CMT disease has no cure now,” Oh said. “If approved, this could be a first-in-class drug.” He added that the data will be presented again at the Hong Kong Society for Stem Cell Research in May with updated follow-up results.
Director Oh explained that EN001 has a competitive edge from cell selection to culture.
“When we isolate cells from the umbilical cord, we go through a screening process that analyzes the mother’s history and the cord itself to identify protein markers and mRNA,” he said. “In the culturing process, we mimic the microenvironment of the umbilical cord, which allows the cells to proliferate faster.”
“As a result, we get younger and more active cells,” Oh said. “We’ve seen better efficacy in animal studies than stem cell therapies.”
Oh attached significance to the recent approval by the U.S. Food and Drug Administration (FDA) of Australian biotech Mesoblast’s allogeneic mesenchymal stem cell therapy Ryoncil.
“This is the first time the FDA has approved a mesenchymal stem cell-based therapy,” Oh said. “The FDA is very demanding regarding CMC (chemistry, manufacturing, and quality control) for cellular therapies, and this approval establishes a pathway through the regulatory hurdles. It opens the door to major markets.”
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