국가임상시험지원재단

● Gene and Cell Therapy: How It Started, Where We Are, and What Is Next?

?Young Jik Kwon(University of California)

Among many modes of emerging therapies, gene and cell therapy has been of great interest recently as fully approved products start to step in the market. They share common technological fundamentals: Controlled biological functions via the molecular manipulation of genetic information, meaning that both therapies face similar challenges and need common breakthroughs in basic science. Despite tremendous potential impact on healthcare, the race in the field is still on going for several decades and many multi- dimensional barriers for clinical translation exist. In this talk, the beginning of the gene and cell therapy, key milestones and drawbacks in history, current status of the technology, and perspectives of the technology will be introduced and discussed, particularly in context of clinical translation and commercialization. By providing a macro view of the topic, it will allow the audience to grasp the insightful ideas about the exciting technology with uncertain fate.  

● Current Status of Gene Therapy

Jae-Gyun Jeong(ViroMed Co., Ltd.)

Since the first human trial for gene therapy was conducted in 1989, 2,759 trials had been carried out for various diseases as of Nov 2017. Among them, a handful of products successfully entered the market. The first was “Glybera” which was approved in 2012 by the European regulatory authority, EMA. This product targeted a rare disease, lipoprotein lipase deficiency and gained huge attention because of several facts; the first gene therapy product marketed in advanced countries, the real proof showing the efficacy of gene therapy, and the most expensive drug in the world (its price tag was over 1 million dollars). Before we saw the first success story in this field, a tremendous number of trials experienced failures and frustration due to the lack of efficacy or severe adverse events. 2017 was another landmark year for gene therapy. Three new gene therapy products in the US and one product in Korea were approved. Still, there are people raising questions about whether this technology can guarantee both sufficient efficacy and safety. During my talk, I will focus on the features of gene therapy products currently available in the market and how they can provide treatment options to patients with unmet medical needs. 

● Current Status of Gene Therapy

Kyungtak Nam(MFDS)

Regenerative medicine is a field that enables radical treatment by restoring human function by treating, replacing or regenerating damaged tissues and organs, and has been attracting attention as a technique to treat patients who are difficult to treat with current therapies such as rare incurable diseases.
In this presentation, I will introduce the status of the development of cell therapy and gene therapy products in Korea and the status of the related laws and regulations.
The commercialization of cell therapy products in Korea has more than 10 years of experience, and it is strong in that the government has a strong will to support the industry and has accumulated a lot of know-how about commercialization.
On the other hand, the biotechnology applied in the field of regenerative medicine is rapidly developing, but the regulation is limited in the traditional and comprehensive framework of medical law and pharmaceutical law, which does not reflect the specificity of regenerative medicine.
Advanced regenerative medicine and advanced biopharmaceuticals are developed in patient-customized form using living cells or tissues. Unlike traditional chemical drug, there are limitations in demonstrating safety and efficacy through animal experiments.
To reflect these considerations, the government will enact a 'Law on Advanced Regenerative Medicine and Advanced Biopharmaceuticals' that stipulates all the necessary items for safety management and support required for each phase of research and development of advanced biopharmaceuticals
In this presentation, I will introduce the status of the development of cell therapy and gene therapy products in Korea and the status of the related laws and regulations.? 

1. Young Jik Kwon(University of California)

2. Jae-Gyun Jeong(ViroMed Co., Ltd.)

3. Kyungtak Nam(MFDS)

상기 과정은 보수과정으로 직능별, 단계별 인정가능시간이 상이함으로 관련규정 '[별표1] 임상시험등 종사자별 교육과정 및 이수시간'을 꼭 확인하시기 바랍니다.

[별표 1]

임상시험등 종사자별 교육과정 및 이수시간

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