초록 ▼

○ 본 연구에서는 골관절염, 외상 등으로 관절연골 결손이 있는 대상자에 대해 자가유래 연골세포치료제(카티라이프) 이식과 미세천공술 간의 유효성 및 안전성을 비교하기 위한 다기관,활성대조군, 공개, 제2상 임상시험을 실시하여(시험군 20명,대조군 10명)카티라이프 이식과 미세골절술 간의 유효성(MRI MOCART scoring, Lysholm score, IKDC score, KOOS, 100mm Pain VAS 등) 및 안전성 차이를 비교 검정함
○ 또한 최종목표인 조건부 품목허가를 위한 품질(CMC), 안전성·유효성 자료를

○ 본 연구에서는 골관절염, 외상 등으로 관절연골 결손이 있는 대상자에 대해 자가유래 연골세포치료제(카티라이프) 이식과 미세천공술 간의 유효성 및 안전성을 비교하기 위한 다기관,활성대조군, 공개, 제2상 임상시험을 실시하여(시험군 20명,대조군 10명)카티라이프 이식과 미세골절술 간의 유효성(MRI MOCART scoring, Lysholm score, IKDC score, KOOS, 100mm Pain VAS 등) 및 안전성 차이를 비교 검정함
○ 또한 최종목표인 조건부 품목허가를 위한 품질(CMC), 안전성·유효성 자료를 국제공통기술문서(CTD) 양식으로 작성하였고, GMP, 위해성관리계획 수립하여 연골 결손에 대한 근본적 재생을 위한 세포치료제로서 무지지체 조직공학 기술을 이용한 구슬형 바이오 인공연골 카티라이프의 조건부 품목허가를 득함
○ 본 연구과제의 수행중 SCI 논문 1편이 게재되었으며, 품목허가 1건의 실적을 달성하였음. 또한 8인의 신규 고용창출의 실적을 달성함
○ 무지지체 조직공학 기술의 카티라이프는 작은 구슬형으로 최소 침습적 이식으로 수술 시간이 짧고 빠른 회복을 기대할 수 있으며, 실제 연골조직과 구조적, 기능적으로 유사하여 이식 즉시 직접적인 구조적 복구와 물리적지지 기능을 발휘함.

(출처 : 요약서 3p)

Abstract ▼

□ Purpose & Contents
Perform phase 2 clinical trial of CartiLife, a cell therapy product for the regeneration of cartilage lesions using scaffold-free tissue engineered bead-type artificial cartilage. The purpose is to establish clinical efficacy, clinical safety and receive conditional approval

□ Purpose & Contents
Perform phase 2 clinical trial of CartiLife, a cell therapy product for the regeneration of cartilage lesions using scaffold-free tissue engineered bead-type artificial cartilage. The purpose is to establish clinical efficacy, clinical safety and receive conditional approval for authorization
○ Recruit and register clinical trial subjects (Test group, 20; Active control group, 10)
○ Investigational product’s manufacture and quality control
○ Transplantation of investigational product or implementation of microfracture procedures on respective groups
○ Follow-up observation on clinical trial subjects
- Efficacy : MRI (MOCART Scoring), Lysholm score, IKDC score, KOOS, 100mm Pain VAS, etc.
- Safety : Physical examination, adverse event, vital signs, laboratory tests, MRI (out-growth)
○ Complete CTDs of CMC and non-clinical·clinical data
○ Preparation of documentation for GMP evaluation
○ Establish a Risk Management Plan (RMP)
○ Submission and approval of product approval application after reviewing data for non-clinical·clinical data and quality (CMC)

□ Results
○ Completion of phase 2 clinical trial.
○ New Drug Approval (2019.04.24.)
○ Publication : Publication of SCI paper related to chondrocyte expansion technique for CartiLife. Revision of SCI paper related to CartiLife’s clinical trial results is in progress.
○ Academic Presentation : Presentation of the results of the clinical trials for CartiLife at the Korea Society of Stem Cell and Regenerative Medicine for Locomotor System, International Symposium for Cell Therapy, Japanese-Korean Combined Orthopaedic Symphosium (2016), World Congress of Regenerative Medicine and Stem Cell (2017), Korean Society of Cartilage and Osteoarthritis, World Congress on Osteoarthritis, International Cartilage Repair Society etc. (2018) and International Cartilage and Osteoarthritis Symposium, Korean-Japanese Combined Orthopaedic Symposium etc. (2019).
○ Hire of new workforces : Hiring 7 manufacturing and QC personnel in preparation for full-fledged sales.

□ Expected Contribution
○ Technological Aspects
- The commercialization of CartiLife will allow us to achieve superiority over first world countries in the scaffold-free tissue engineering technology sector, with a basis on tissue stem cells through a successful product commercialization.
- Autologous extracellular matrix provides micro-environment to the cells and its autologous character prevents the need for a xenogeneic/synthetic scaffolds which may be a cause of inflammation and immune responses after transplantation. This may be used as a ‘next generation’ tissue engineering technology.
- The product’s small pellet morphology allows for the permeation of nutrients thorough the tissue to the core. This technology may not only be utilized for tissue cultures, but also as a means to increase the cell’s survivability in transplantation procedures.
- The technology can be exported as a next-generation cartilage regeneration technology as the company owns the patent.
○ Economical and Industrial aspect
- By expanding clinical application through the industrialization of the ideal therapy, we can expect the fast and accurate treatment of cartilage defects. We will also be able to contribute the promotion of public health and bring substantial increase to the Quality of Life through predictable decreases in medical expenses, namely through the reduction of a requisite hospitalization period as well as through the reduction in treatment costs and need for re-treatment procedures.
- Since CartiLife is capable of treating not only younger patients with small cartilage defects but also older patients with larger cartilage defects, it will be able to target both the cell therapy market as well as the artificial joint market to generate an import-substitution effect.
- CartiLife will contribute to an increase in job opportunities through the recruitment of specialized workers into the manufacture, QC/QA, clinical trials and others as valuable industrial human resources in the regenerative medicine field.

(출처 : SUMMARY 6p)

목차 Contents

• 표지 ... 1
• 제출문 ... 2
• 보고서 요약서 ... 3
• 국문 요약문 ... 4
• SUMMARY ... 6
• 목차 ... 8
• 1. 연구개발과제의 개요 ... 9
• 1-1. 연구개발 목적 ... 9
• 1-2. 연구개발의 필요성 ... 9
• 1-3. 연구개발 범위 ... 12
• 2. 국내외 기술개발 현황 ... 15
• 3. 연구수행 내용 및 결과 ... 19
• 3-1. 연구 수행 결과 ... 19
• 3-2. 연구개발 추진전략·방법 ... 27
• 3-3. 연구개발성과 ... 27
• 3-4. 기술적 성과 ... 31
• 3-5. 경제적 성과 ... 33
• 4. 목표달성도 및 관련분야 기여도 ... 34
• 4-1. 목표달성도 ... 34
• 4-2. 관련분야 기여도 ... 35
• 5. 연구결과의 활용계획 ... 36
• 5-1. 연구결과물의 활용계획 ... 36
• 5-2. 연구 결과물의 사업화 계획 ... 36
• 6. 연구과정에서 수집한 해외과학기술정보 ... 38
• 7. 연구개발성과의 보안등급 ... 39
• 8. 국가과학기술종합정보시스템에 등록한 연구시설·장비 현황 ... 39
• 9. 연구개발과제 수행에 따른 연구실 등의 안전조치 이행실적 ... 39
• 10. 연구개발과제의 대표적 연구실적 ... 40
• 11. 기타사항 ... 40
• 12. 참고문헌 ... 41
• 별첨4. 실적 증빙자료 ... 43
• 끝페이지 ... 49