"Cure diseases with cells and genes." Cell and gene therapy (CGT), which had been confined to lab experiments, is emerging as the key to overcoming the limits of treating intractable and rare diseases and as a future growth engine for the global pharmaceutical and biotech industry.
CGT is a new-concept medicine that fundamentally cures diseases by modifying a patient's cells or genes. It uses living autologous or allogeneic cells to restore cellular and tissue function, and it works by replacing abnormal genes with normal ones or adding new functions to treat genetic diseases.
The greatest value of CGT is that it can deliver long-term effects with a single treatment. Unlike existing therapies that must be taken for years or administered periodically, it directly edits a patient's genes or replaces damaged tissue with normal tissue to fundamentally treat disease—a so-called "one-shot cure."
This is not a far-off story. The number of CGT approvals is already increasing, mainly in the United States and Europe, and the scope of treatment (indications) is expanding. According to BioInformant, a global biotechnology analytics firm, as of May this year, the U.S. Food and Drug Administration (FDA) has approved a total of 43 cell and gene therapies.
◇"Diseases with no answers aim for a cure with one dose"
AMTAGVI by Iovance Biotherapeutics is a representative cell and gene therapy approved by the FDA last year. AMTAGVI received accelerated approval as a treatment for adult melanoma (skin cancer) patients whose disease is unresectable by surgery or has metastasized to other parts of the body after existing anticancer treatments, becoming the world's first T-cell therapy for solid tumors.
T cells are immune cells that eliminate or prevent the proliferation of cells infected by pathogens. They also help B cells that produce antibodies. AMTAGVI works by isolating T cells from a patient's tumor tissue, expanding them ex vivo, and reinfusing them into the patient, enabling immune T cells to attack cancer cells.
At the American Society of Clinical Oncology (ASCO) annual meeting in July this year, researchers presented five-year follow-up results from 153 patients with advanced melanoma who participated in the phase 2 trial of AMTAGVI. The objective response rate (ORR)—the proportion of patients whose tumors shrank noticeably—was 31.4%, and the five-year overall survival (OS) rate was 19.7%.
Simply put, among melanoma patients unresponsive to existing anticancer drugs, one in three saw their tumors shrink or disappear completely with just a single dose of AMTAGVI, and one in five survived for more than five years after receiving AMTAGVI.
Orchard Therapeutics in the United Kingdom also won FDA approval in March last year for the gene therapy Lenmeldy to treat metachromatic leukodystrophy (MLD) in children. MLD is a genetic disease caused by a deficiency of the enzyme arylsulfatase A (ARSA) that severely affects the brain and nervous system, causing paralysis, gait disturbance, and intellectual disability. With no effective treatment to date, patients have been known to die within three to four years of onset.
Lenmeldy is a therapy that corrects the genetic defect that causes MLD. It uses a harmless lentivirus as a vector to deliver the therapeutic gene. A viral vector carrying the ARSA gene is delivered to stem cells extracted from the patient's body; when these are reinfused, the ARSA enzyme is produced normally, halting or slowing disease progression.
◇"From technology to industry" the CGT market is growing
Because CGT tackles medicine's limits and toughest challenges, its development journey is arduous, but success is expected to create a large market. According to EvaluatePharma, the global CGT market is projected to grow at a compound annual rate of 45.7%, from about $6.5 billion (about 9 trillion won) in 2021 to about $89 billion (about 127 trillion won) in 2028.
Global big pharma such as Novartis of Switzerland, Johnson & Johnson (J&J) of the United States, and Bristol-Myers Squibb (BMS) of the United States entered the field early and succeeded in commercializing next-generation anticancer chimeric antigen receptor (CAR) T-cell therapies.
AstraZeneca in the United Kingdom this year acquired Belgian gene therapy specialist EsoBiotec for up to $1 billion (about 1.4 trillion won). Bayer of Germany acquired BlueRock Therapeutics, which develops induced pluripotent stem cell (iPS cell)-based therapies, in 2019 and AskBio, which owns an adeno-associated virus (AAV)-based gene therapy platform, in 2020.
In Korea, CHA Biotech, Pharmicell, Kimcell&Gene, MEDIPOST, S.Biomedics, ENCell, GC Cell, Kolon TissueGene, and HLB Innovation have entered CGT development.
One of the main topics at the Healthcare Innovation Forum (HIF 2025), to be held on Nov. 6 at the Westin Josun Hotel in Jung District, Seoul, hosted by ChosunBiz and the Korea Health Industry Development Institute (KHIDI), is "regenerative medicine technology." Attendees will be able to meet renowned experts in this field on site.
First, Cha Kwang-ryeol, head of the Global Research Institute at CHA Hospital and CHA Bio Group, who has gained global recognition in infertility, stem cells, and regenerative medicine, will deliver a keynote speech on "the importance of K-cell therapy." CHA Bio Group is building a combined cell and gene therapy facility (CGB) in the second Pangyo Techno Valley. CGB will cover 66,115 square meters (about 20,000 pyeong) and begin operations in 2027.
Kim Hyo-su, founder and CEO of Kimcell&Gene and a professor at Seoul National University Hospital's Biomedical Research Institute, will give a lecture on "new developments in gene therapy: development of cardiovascular therapies based on cellular reprogramming." Eom Hyeon-seok, head of the full-cycle technology development group for immune cell gene therapy at the National Cancer Center, will present "development of next-generation immune cell gene therapy: achievements in hematologic cancers and expansion to solid tumors."
Pharmicell CEO Kim Hyun-su, who commercialized the world's first stem cell therapy, will give a lecture on "a new era created by stem cell therapies." Kim previously served as a professor of hematology-oncology at Ajou University Hospital and currently runs a hospital specializing in stem cell therapy together with Pharmicell.
◎ 2025 Healthcare Innovation Forum overview
△Date and time: Thu., Nov. 6, 2025, 9 a.m.–4:20 p.m.
△Venue: Grand Ballroom, Westin Josun Hotel, Sogong-dong, Seoul
△Theme: AI and advanced regeneration, crossing the boundaries of healthcare
△Hosted by: Korea Health Industry Development Institute (KHIDI), ChosunBiz
△Supported by: Ministry of Health and Welfare
△Registration and fees: https://e.chosunbiz.com
△Inquiries: 02-724-6157, event@chosunbiz.com