Pharmaceutical and Biotech Industries Welcome Amendments to the Advanced Regenerative Medicine Act — but What Should Be Considered?

Cell and Gene Therapies Can Now Be Administered to Patients Through Clinical Research
Safety and Quality Control Remain Key Challenges — “Greater Role Expected from the Review Committee”

GC Cell Research Institute Director Won Seong-yong (right) speaks during the regenerative medicine session at BIO KOREA 2024, held at COEX in Seoul on the 10th.

Expectations are rising across Korea’s pharmaceutical and biotechnology industry following the passage in February of amendments to the Act on Safety of and Support for Advanced Regenerative Medicine and Advanced Biopharmaceuticals, commonly referred to as the Advanced Regenerative Medicine Act. The revisions are expected to stimulate regenerative medicine and biopharmaceutical research and accelerate the development of cell and gene therapies (CGTs).

Previously, investigational advanced regenerative medicine treatments were generally limited to patients with rare or refractory diseases who were participating in clinical trials. Under the amended law, however, treatment opportunities may also become available to patients outside conventional clinical trials through advanced regenerative medicine clinical research. Companies including CHA Biotech, GI Cell, CGBio, and NKMAX have publicly welcomed the changes.

Against this backdrop, a panel discussion was held during BIO KOREA 2024 at COEX in Seoul on the 10th to discuss industry expectations and the outlook following the amendment. Panelists included Won Seong-yong, Director of the GC Cell Research Institute; Son Hyun-jung, Head of Clinical Development at Vaxcell-Bio; Hong Sung-sang, Executive Director at ENCell; and Lee Joo-yeon, Research Institute Director at Xcell Therapeutics.

Won said the amendment represents “an opportunity to provide advanced biopharmaceuticals to more patients while also accelerating therapeutic development.” He added, “We expect the advanced biopharmaceutical market to expand further as patient populations and indications become more precisely segmented.”

Son said that expanding access to regenerative medicine could also create revenue opportunities through the supply of therapeutic products. “For companies like ours, which need to generate revenue within three years after listing, this is a positive development,” she said. “Unlike commercial clinical trial data, results from ‘clinical research’ cannot be used directly for marketing authorization applications, but they can still provide safety data at relatively low cost.”

Hong noted that companies or research institutions without their own cell processing facilities cannot manufacture advanced biopharmaceuticals independently. “As a result, more investigational products are likely to be outsourced to contract development and manufacturing organizations (CDMOs), which should naturally lead to increased revenue for advanced biopharmaceutical CDMO companies,” he said.

Lee added, “Despite the growth of the advanced biopharmaceutical market, regulatory approval has remained a significant hurdle. As those barriers are eased, we expect growth in supporting industries—including materials, components, and equipment required for advanced biopharmaceutical development and manufacturing. There is also growing interest in the domestic development of culture media for biopharmaceutical production.”

From left: Lee Joo-yeon, Research Institute Director at Xcell Therapeutics; Hong Sung-sang, Executive Director at ENCell; Son Hyun-jung, Head of Clinical Development at Vaxcell-Bio; Won Seong-yong, Research Institute Director at GC Cell; and Hwang Yoo-kyung, CEO of YUYA Bio Consulting.
Photo courtesy of GC Cell

At the same time, the panelists pointed to several challenges that the industry must address collectively. They stressed that the broader availability of regenerative medicine following the amendment should not lead to indiscriminate research or treatment, and that companies must strengthen product safety and quality management. There were also calls for a more active role by the Advanced Regenerative Medicine and Advanced Biopharmaceuticals Review Committee under the Ministry of Health and Welfare.

Won Seong-yong said, “The area I am most concerned about is safety. Because these products are administered to humans, it is essential to establish not only QC (quality control) but also a robust CMC framework covering chemistry, manufacturing, and controls. At our company, we have adopted a strategy of prioritizing pipelines that have already demonstrated safety in clinical trials or have received IND approval before moving them into clinical research.”

He added, “We also need clear standards defining what level of efficacy should be required before a product can be used for therapeutic purposes. I believe the Review Committee will establish regulations at a level that both the industry and patients can reasonably understand.”

Son Hyun-jung also agreed on the importance of quality control. “The Review Committee needs to manage quality issues rigorously,” she said. “The challenge is that some companies may not be able to keep pace quickly enough with legal amendments or tighter regulatory requirements. In such cases, it is important to assess the company’s readiness with the help of external consulting firms or legal and regulatory experts.”