"Cell and gene therapies are fields that global pharmaceutical corporations cannot afford to give up. The global cell and gene therapy market will continue to grow significantly."
Jang Jong-wook, CEO of ENCell, met with ChosunBiz at ENCell's headquarters in Gangnam, Seoul, on the 3rd, and said, "It is the mission of pharmaceutical corporations to save patients who cannot be treated with existing therapies, so the challenge of developing cell and gene therapies will continue."
Cell and gene therapy (CGT) is a third-generation biopharmaceutical that fundamentally treats diseases by modifying or transforming patients' genes or cells, unlike traditional medicines. It is considered the key to treating difficult and rare diseases that have been hard to address with existing therapies. In fact, several global pharmaceutical giants, including Novartis, Gilead, and Janssen, have invested in this field and successfully commercialized drugs.
According to the global market research firm Frost & Sullivan, the scale of the cell and gene therapy market is expected to grow more than threefold, from $16.33 billion in 2023 (approximately 23.8 trillion won) to $55.59 billion in 2026 (approximately 81.39 trillion won).
ENCell is a spinoff company founded in March 2018 by Professor Jang Jong-wook of Samsung Seoul Hospital's Future Medicine Research Center and was listed on the KOSDAQ in August last year. The company's business mainly consists of contract development and manufacturing of cell and gene therapies (CDMO) and new drug development. It aims for a virtuous cycle by reinvesting profits gained from the CDMO business into new drug development.
◇ "Expanding sales through diversification of cell and gene therapy products"
ENCell holds the top position in domestic contract development and manufacturing of cell and gene therapies. The key to success lies in having anticipated the opening of the cell and gene therapy market early and establishing business infrastructure to secure competitiveness. The company is the only one in the country equipped with certified facilities (GMP) capable of producing both cells and viruses simultaneously. Based on this, it has succeeded in securing continuous contracts for contract manufacturing of cell therapies from major global pharmaceutical corporations.
ENCell is responsible for the production of leading CAR-T therapies such as Novartis' Kymriah and Janssen's Carvykti. CAR-T therapies involve extracting T cells, which are a type of immune cell from patients, and modifying the genes to target cancer cells. This allows for targeting only cancer cells while leaving normal cells unaffected, thereby enhancing treatment efficacy and reducing side effects.
Jang said, "Since the establishment of ENCell, we had the technology and facilities to engage in CDMO business, allowing us to receive contract manufacturing requests immediately when cell and gene therapies first entered the market."
He explained that "ENCell has a diversified small-scale production system specialized in cell and gene therapies," adding, "While large-scale production is crucial for second-generation biopharmaceuticals, it is essential for third-generation biopharmaceuticals, such as cell and gene therapies, to produce various treatments in smaller quantities to provide tailored care to multiple patients." He noted that large CDMO corporations like Samsung Biologics and Lonza focus on large-scale production of second-generation biopharmaceuticals, while ENCell began its business with a focus on the market for third-generation cell and gene therapies.
ENCell has targeted the adeno-associated virus (AAV) gene therapy market as a way to strengthen its CDMO competitiveness. This gene therapy operates on the principle of delivering therapeutic genes to cells via AAV to correct abnormal genes, with ENCell responsible for AAV production. A representative AAV gene therapy is Novartis' Zolgensma for treating spinal muscular atrophy, which exceeded 1 trillion won in sales just one year after its launch in 2020.
Earlier this year, ENCell also completed the development of AAV production processes with Sartorius Korea, the Korean branch of Sartorius. Jang said, "AAV production requires advanced technology in virus vector production, purification processes, and analysis, and ENCell has the technology and infrastructure to handle it," adding that "this year, ENCell plans to diversify its portfolio into gene therapies to boost CDMO sales."
◇ "Encouraging clinical developments for new drug development... enhancing momentum for advanced regenerative medicine laws"
Another major focus of ENCell's business is new drug development. It is currently developing the cell and gene therapy EN001 targeting treatments for muscular diseases, including Charcot-Marie-Tooth disease, known as a hereditary disorder of the Samsung family, as well as Duchenne muscular dystrophy and cachexia.
EN001 is a mesenchymal stem cell therapy candidate being developed by ENCell, produced from mesenchymal stem cells obtained in the early culture stages.
The clinical trial for the treatment of Charcot-Marie-Tooth disease (CMT) is progressing the fastest, with recent meaningful results indicating a higher likelihood of success in new drug development.
Charcot-Marie-Tooth disease is a hereditary neuropathy that affects peripheral nerves, and there are currently no treatments available. Non-clinical research has confirmed nerve and muscle regeneration, completing Phase 1 trials on type 1A CMT patients in June 2023. Type 1A CMT accounts for 50% of all CMT patients. Clinical trials have also been conducted on the other type, CMT 1E. The research team previously reported that all study subjects experienced no major side effects during the clinical study period, and some patients showed significantly improved clinical symptoms.
Jang stated, "CMT 1A has been conducted as a commercial clinical trial, while CMT 1E has been carried out as an advanced regenerative clinical trial, and both disease groups showed improvements in symptoms based on neuropathic pain scales." He noted that "efficacy was confirmed 16 weeks after administration compared to the baseline," adding, "Moreover, no matter how good the treatment effect is, if the toxicity is high, it cannot be used, so evaluating side effects and safety is crucial, and we have secured safety indicators, enabling numerous future challenges."
ENCell began repeated dosing for high-dose groups in Phase 1b clinical trials in December last year. After confirming safety and exploratory therapeutic effects in the low-dose group of EN001, the goal is to confirm the same level of effect in the high-dose group, which has double the dosage. The plan is to complete patient dosing within the year and generate results to expedite Phase 2 trials.
Jang noted that with the recent implementation of the amendment to the "Act on the Safety and Support of Advanced Regenerative Medicine and Advanced Biopharmaceuticals" (hereinafter referred to as the advanced regenerative medicine law), opportunities for not only new drug development but also CDMO business are expected to increase.
The advanced regenerative medicine law is centered on revising regulations to allow the use of cell and gene therapies at the developmental stage, which had previously been restricted to research purposes only for diseases with no available treatments or rare and difficult diseases. The ease of linking university hospital clinical trials with corporate technologies is also one of ENCell's competitive advantages.
Jang said, "First of all, since EN001 is already in commercial clinical trials and investigator-initiated clinical trials, once the advanced regenerative medicine law is enacted, we will be in a position to immediately submit treatment plans for patient-targeted therapies," adding that "the implementation of this law will further accelerate the development of new drugs for patient treatment."
He further emphasized that "since patients with rare and difficult diseases will be able to use unapproved medicines for treatment purposes, it will create opportunities for contract manufacturing of new drugs, therefore enhancing the growth of the CDMO business," and that ENCell will put more effort into strengthening its CDMO business and increasing the success rate of new drug development.