It felt like a mix of joy and burden as the diagnosis rate for rare and intractable diseases improved, but the lack of available treatments left the feeling of indebted to patients and couldn’t help. This led to the creation of the G-CROWN platform project to develop treatments directly.
Samsung Medical Center Launches South Korea’s Pioneering Gene Therapy Development Platform Project
Dr. Lee Ji-hoon, director of the Gene Therapy Research Center at Samsung Medical Center and professor of pediatrics, explained on April 22 the origins of the country’s first open platform project for developing cutting-edge gene therapies for rare and intractable diseases (G-CROWN platform).
Globally, experts estimate over 8,500 types of rare diseases exist, yet less than 5% have available treatments. Even when treatments are developed, cost remains a significant hurdle. For example, Zolgensma, a gene therapy for spinal muscular atrophy, costs about 2 billion KRW (approximately 1.35 million USD) for a single dose. Other ongoing treatments can run into hundreds of millions of KRW annually. While patients shoulder a substantial portion of these expenses, health insurance covers costs beyond a certain threshold.
The core issue is that most major gene therapies are developed overseas and imported into South Korea. As more imported treatments become available, the financial burden on both individuals and the state continues to grow.
To address these challenges, Samsung Medical Center has launched the G-CROWN platform, integrating the entire gene therapy development process. This long-term project, backed by the Ministry of Health and Welfare’s research-focused hospital research and development (R&D) initiative, began in 2022 with a planned investment of 388 billion KRW (approximately 263 million USD) by 2030. It involves collaboration between hospitals, universities, and private companies.
The Hospital Spearheads Research and Business Development
The G-CROWN platform’s key innovation is unifying the gene therapy development process into a seamless workflow. Previously, basic research, preclinical studies, clinical trials, and commercialization were separate stages. Now, they’re integrated and driven by patient data.
Notably, this initiative is hospital-led, marking the first time a South Korean hospital has directly engaged in gene therapy development. The research team guides drug development direction and actively participates in clinical trial design, leveraging patient data and clinical expertise.
Dr. Lee emphasized that developing gene therapies demands cutting-edge technology and accurately predicting patient responses is crucial. He noted that the hospital possesses both a structured database and the clinical experience necessary to validate these responses.
The research team creates disease models using patient-derived cells to pre-validate potential therapeutic candidates’ effectiveness. This process then progresses to animal testing and clinical trials.
He explained that by first confirming efficacy in patient cell-based models, they can significantly reduce the likelihood of failure. The platform aims to systematize this crucial validation process.
Patient Data-Driven Approach Accelerates Clinical Trials
The hospital-centered platform’s strengths become particularly evident during clinical trials. Rare disease trials face unique challenges due to small patient populations, making recruitment difficult. Lack of trial design experience often leads to failures.
Samsung Medical Center has developed a database of clinical cohorts for rare disease patients, enabling rapid recruitment of initial trial subjects. An A-CRO team and specialized medical staff support the entire process, from establishing clinical protocols to trial execution.
Alongside platform development, actual treatment creation is underway. The team is collaborating with companies to develop adeno-associated virus (AAV) vectors that efficiently deliver genes across muscle tissue for Duchenne muscular dystrophy. They’re also selecting genes to maximize therapeutic effects. After validating efficacy in patient-derived muscle cell models, they’ll advance to animal testing.
Hereditary retinal diseases show the most rapid progress. The team has developed retina-specific vectors and is currently evaluating therapeutic candidates in animal trials.
The research team aims to begin clinical trials around 2028. As the platform matures, they expect drug development timelines to shorten.
Patient-Derived Cell Models Tackle Hepatotoxicity and Gene Delivery Efficiency Challenges
While gene therapies show promise, they still face high failure rates and technical hurdles. Issues with gene delivery efficiency, hepatotoxicity at high doses, and immune responses have led even major pharmaceutical companies to pause or slow some projects.
Dr. Lee noted that global failures often stem from low delivery efficiency and immune responses. He explained that their platform focuses on analyzing, validating, and addressing these factors proactively. For instance, they’re developing liver-bypassing therapies to eliminate hepatotoxicity and exploring immunosuppressant use to mitigate immune responses.
However, with each gene therapy costing tens of billions of KRW to develop, securing additional funding remains a challenge for domestic drug development. Nevertheless, this platform represents more than just R&D; it’s an attempt to transform rare disease treatment in South Korea. The goal is to shift from reliance on foreign treatments to building domestic capabilities, ultimately reducing costs and boosting industrial competitiveness.
Dr. Lee concluded that its moving beyond importing foreign-developed treatments to creating and applying its own. The G-CROWN platform aims to commercialize these gene therapies, generate profits, and reinvest in further development, creating a sustainable cycle.
He added that by 2030, it aims to develop at least three in vivo and five ex vivo gene therapies. The goal is to create treatments for rare and intractable diseases that aren’t available elsewhere, ensuring South Korean patients benefit first from these breakthrough therapies.
