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Hong Kong & Guangzhou International Conference on Stem Cell & Regenerative Medicine
16 December 2016, Friday Charles K. Kao Auditorium, Hong Kong Science Park
PROPOSED Programme Rundown
(Draft as at 13 December 2016)
Please CLICK HERE to register for the conference.
09:00 – 09:30 Registration
09:30 – 09:55 Welcome Remarks The Honourable Mrs Fanny Law, GBS, JP Chairperson, Hong Kong Science and Technology Parks Corporation
Opening Remarks by Guest-of-Honour The Honourable C Y Leung, GBM, GBS, JP The Chief Executive of the Hong Kong Special Administrative Region
Speech by Guest-of-Honour Prof. Bai Chunli President, Chinese Academy of Sciences
Speech by Guest-of-Honour Prof. Pei Duanqing, Professor and Director General, Guangzhou Institutes of Biomedicine and Health (GIBH), Chinese Academy of Sciences
Speech by Guest-of-Honour Mr Michael T. Murphy, Chief Development Officer, The National Academies of Sciences, Engineering and Medicine, US
09:55 – 10:15 Current Regulatory Landscape – Similarities and Differences Prof. Marc Turner Medical Director, Scottish National Blood Transfusion Service, Scotland
Regulatory Principles 10:15 – 10:45
Operational Challenges in the Manufacture of Cellular Therapies Prof. Marc Turner Medical Director, Scottish National Blood Transfusion Service, Scotland
10:45 – 11:15 Networking Coffee Break / Mini-Exhibition
11:15 – 11:45 The Regulatory Landscape for Cell and Gene Medicinal Products in the EU Dr Jacqueline Barry Director of Regulatory Affairs, Cell and Gene Therapy Catapult, UK
11:45 – 12:15 The Ethics and Science of Stem Cell Research and Therapy Prof. Martin Pera Professor of Stem Cell Sciences, University of Melbourne
12:15 – 14:00 Networking Lunch / Mini-Exhibition
Clinical Applications 14:00 – 14:30 Stem Cell Therapy for Liver Fibrosis
Prof. Philip Newsome Director of Centre for Liver Research/ Professor of Hepatology University of Birmingham
14:30 – 15:00 Cell Therapy for Diabetes - Clinical and Translational Advances Dr John J Casey Director of the Scottish National Islet Transplant Programme and Chair UK Pancreas Transplant Advisory Group University of Edinburgh/ Royal Infirmary of Edinburgh
15:00 – 15:30
Illuminating Alzheimer’s Disease through Epigenetic Fingerprinting and Tissue Engineering using IPSCs Prof. Li-Huei Tsai Professor and Director, Massachusetts Institute of Technology
15:30 – 15:40
Closing Remarks Prof. Lap-Chee Tsui , GBM, GBS, JP President of The Academy of Sciences of Hong Kong President of Victor and William Fung Foundation
Note: Information is subject to change without prior notice.
Speakers biography & synopsis
Operational Challenges in the Manufacture of Cellular Therapies Prof. Marc Turner Medical Director Scottish National Blood Transfusion Service, Scotland
Biography: Marc Turner is Professor of Cellular Therapy at the University of Edinburgh and Medical Director at the Scottish National Blood Transfusion Service. He qualified in Medicine from the University of Manchester in 1982 and trained in General Medicine and Haematology. He obtained his PhD in human haematopoietic stem cell biology from the University of Edinburgh in 1995 and an MBA in Life Sciences from the Open University in 2007. He is a Fellow of the Royal College of Physicians of London, of the Royal College of Physicians of Edinburgh, of the Royal College of Pathologists and of the Higher Education Academy. His research interests include immunohematology, variant CJD transmission by blood and tissues and translational research in stem cell therapy and adoptive immunotherapy. He has 150 publications and has held 35 research grants. He is a Non-Executive Director of the Cell Therapy Catapult and a clinical member of the EMA Committee on Advanced Therapies.
The Regulatory Landscape for Cell and Gene Medicinal Products in the EU Dr Jacqueline Barry Director of Regulatory Affairs Cell and Gene Therapy Catapult, UK
Biography: Jacqueline Barry is Director of Regulatory Affairs for Cell and Gene Therapy Catapult, the UK’s centre for the acceleration of the translation of cell therapies towards commercialisation. Prior to this Jacqueline worked at the Scottish National Blood Transfusion Service in a number of senior regulatory and quality positions, the responsibility for which included designing the regulatory strategy for the Cellular Therapies developed by the Blood Transfusion Service, acting as Responsible Person for Blood and Qualified Person for medicinal product release. Before that she held a number of academic posts at Edinburgh University studying neuromuscular regeneration. She has considerable experience in the development, translation, clinical trial and approval of cell based medicinal products and therapies. The Ethics and Science of Stem Cell Research and Therapy Prof. Martin Pera Professor of Stem Cell Sciences University of Melbourne
Biography: Martin Pera is Professor of Stem Cell Sciences at the University of Melbourne, the Florey Neuroscience Institute, and the Walter and Eliza Hall Institute for Medical Research. He serves as Program Leader for Stem Cells Australia, the Australian Research Council Special Research Initiative in Stem Cell Sciences. Pera received his BA in English Language and Literature from the College of William and Mary, and his PhD in Pharmacology from George Washington University, and undertook postdoctoral training in the UK at the Institute of Cancer Research and the Imperial Cancer Research Fund. He held independent research positions at the Institute of Cancer Research and the Department of Zoology at Oxford University before joining Monash University in 1996. In 2006 he moved to Los Angeles to take up a position as the Founding Director of the Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research at the University of Southern California. He returned to Melbourne in 2011. Pera has provided extensive advice to state, national and international regulatory authorities on the scientific background to human stem cell research. He and his colleagues are currently leading a campaign against the provision of unproven autologous stem cell treatments in Australia.
Stem Cell Therapy for Liver Fibrosis Prof. Philip Newsome Director of Centre for Liver Research/ Professor of Hepatology University of Birmingham
Biography: Philip Newsome is Research Area Lead for Liver Regeneration and Training Lead on NIHR Liver BRU. He is a Scientific Member of the Governing Board of the European Association for the Study of Liver. He runs the metabolic services at the Liver Unit at the Queen Elizabeth Hospital Birmingham which includes a large multi-disciplinary clinic for patients with NAFLD. Professor Newsome has published over 80 research papers in scientific journals as well as many book chapters in the field of Non-Alcoholic Fatty Liver Disease (NAFLD). He was Chief Investigator on a randomised controlled trial of Glucagon-like peptide-1 (GLP-1) therapy in NAFLD published in the Lancet, and is also Chief Investigator on a study of Fibroscan in NAFLD. He is the Co-ordinating Investigator for three global NAFLD studies and Chief Investigator on a UK NAFLD study. He is editor of a recently published textbook on Liver Transplantation, and chaired the national guidelines for liver transplantation in NAFLD. He sits on the NICE Guideline Development Group for NAFLD. He has a large laboratory group focussing on the role of cell therapy in liver injury. He has received a range of funding including large project/programme grants (EUFP7, MRC and MRC/Wellcome Clinical Training Fellowships). He has established two cutting edge clinical trials which he is the Chief Investigator on. One of these, REALISTIC, is the largest clinical trial of haematopoietic stem cell therapy in patients with liver cirrhosis in Europe/US. He co-ordinates the MERLIN (EU FP7) consortium which includes a clinical trial of mesenchymal stromal cells in patients with primary sclerosing cholangitis. He has driven stem cell research at the University of Birmingham resulting in significant strategic investment in manufacturing facilities such as the Advanced Therapies Facility. The success and significance of his work has led to many approaches from industry such that he has an extensive interaction with many bio-tech companies, ranging from provision of consultancy through to joint research projects. Synopsis: Stem Cell Therapy for Liver Fibrosis Present data on pre-clinical efficacy of haematopoietic stem cells in remodelling liver fibrosis along with the data from a randomised controlled trial in patients with liver cirrhosis.
Cell Therapy for Diabetes - Clinical and Translational Advances Dr John J Casey Director of the Scottish National Islet Transplant Programme and Chair UK Pancreas Transplant Advisory Group University of Edinburgh/ Royal Infirmary of Edinburgh
Biography: Dr John Casey is a Consultant Transplant and General Surgeon at the Royal Infirmary of Edinburgh. He is Director of the Scottish National Islet Transplant Service, which has now performed over 60 islet transplants in Scotland and is now one of the world's largest units. He is Clinical Lead for Organ Transplantation for Scotland, Chair of the NHS Blood & Transplant UK Pancreas Advisory Group and set up and chaired the NHS Blood & Transplant UK Pancreas Islet Task Force. He has a large international collaborative research programme in islet and beta cell transplantation (grant funding >£7 million) and has published widely in the fields of islet transplantation and regenerative medicine, organ transplantation and laparoscopic surgery. Synopsis: Cell Therapy for Diabetes - Clinical and Translational Advances Islet transplantation for type 1 diabetes is now established as a clinical service in the UK. The success of this programme demonstrates the efficacy of cell therapy for this chronic disease but relies on donor organs to provide the islets for transplantation. Regenerative strategies are required to expand this successful cell therapy to the expanding patient population. We have been looking at strategies to produce a limitless supply of regenerated human islets for transplantation and overcome some of the technical barriers to translating the science into a clinical programme.
Illuminating Alzheimer’s Disease through Epigenetic Fingerprinting and Tissue Engineering using IPSCs Prof. Li-Huei Tsai Professor and Director Massachusetts Institute of Technology
Biography: Professor Li-Huei Tsai is the Director of the Picower Institute for Learning and Memory at the Massachusetts Institute of Technology, a Picower Professor of Neuroscience, and an Associate Member of the Broad Institute. She obtained Ph.D. from University of Texas Southwestern Medical Center in Dallas and postdoctoral training at Cold Spring Harbor Laboratories and Massachusetts General Hospital. Tsai became Assistant Professor of Pathology at Harvard Medical School and was promoted to tenure Professor at Harvard in 2002. She relocated to Massachusetts Institute of Technology in 2006. She was an Investigator of the Howard Hughes Medical Institute from 1997 to 2013. Tsai is also a Fellow of the American Association for the Advancement of Science, a member of the National Academy of Medicine, and an Academician of the Academia Sinica in Taiwan. Tsai is interested in elucidating the pathogenic mechanisms underlying neurological disorders that impact learning and memory. She takes a multidisciplinary approach to investigate the molecular, systems, and circuit basis of neurodegenerative disorders. Recent contributions include the identification of chromatin remodeling as a means to regulate memory gene expression and enhance cognitive function during neurodegeneration. Her lab also conducts epigenomic analysis of mouse and human Alzheimer’s disease (AD) brain samples and has identified important contributions of dysregulated immune response genes in AD. Currently, the Tsai lab uses induced pluripotent stem cell (iPSCs) derived from human subjects to model AD and large scale imaging, optogenetics, and in vivo electrophysiology to study the brain circuitry affected by AD. Synopsis: Illuminating Alzheimer’s disease through epigenetic fingerprinting and tissue engineering using IPSCs Alzheimer’s disease (AD) is the leading cause of dementia. However, the etiology and molecular mechanisms underlying AD pathogenesis still remain poorly understood. Here, we exploited the advancements in induced pluripotent stem cell (iPSC) technology, and conducted gene expression profiling of AD patient-derived cells at the iPSC stage, and following their differentiation into neural cells. Additionally, by utilizing targeted mass spectrometry, we simultaneously profiled 60 distinct combinatorial histone modifications at the same differentiation stages. Surprisingly, we observed a hitherto unknown stratification of AD, in which familial AD patients harboring mutations in presenilin-1 (PSEN1) display a unique chromatin signature compared to samples from other AD patients and unaffected individuals. While iPSC-based two-dimensional cultures are generally informative, they do not recapitulate the complexity of neural tissue, and phenotypes such as extracellular protein aggregation are difficult to observe. To address these issues, we generated brain organoids that use pluripotent stem cells derived from AD patients and recapitulate AD-like pathologies, including age-dependent amyloid aggregation and hyperphosphorylated tau. Furthermore, treatment of patient-derived organoids with β- and γ-secretase inhibitors significantly reduces amyloid and tau pathology. Together, our epigenetic fingerprinting and tissue engineering efforts could greatly increase the translatability of pre-clinical drug discovery in AD.
Closing Remarks Prof. Lap-Chee Tsui, GBM, GBS, JP President of The Academy of Sciences of Hong Kong President of Victor and William Fung Foundation
Biography: Professor Lap-Chee Tsui is currently President of the Victor and William Fung Foundation, Founding President of the Academy of Sciences of Hong Kong, Director of Zhejiang University’s Qiushi Academy for Advanced Studies, and University of Toronto’s Emeritus University Professor. He is the immediate-past Vice Chancellor of The University of Hong Kong, prior to which, he was Geneticist-in-Chief at the Hospital for Sick Children in Toronto and University Professor at University of Toronto, Canada. He received his Bachelor and Master degrees from the Chinese University of Hong Kong and his PhD from University of Pittsburgh. He is world renowned for his research work in human genetics and genomics, notably the identification of the gene for Cystic Fibrosis and other human genetic diseases while conducting a comprehensive characterization of human chromosome 7. Professor Tsui has over 300 peer-reviewed scientific publications and 65 invited book chapters. He is the recipient of many national/international prizes, and is a Fellow of Royal Society of Canada, Royal Society of London and Academia Sinica. He is a Associate Member of the National Academy of Sciences USA, a Foreign Member of Chinese Academy of Sciences, and is a Canadian Medical Hall of Fame Laureate. His other awards include 15 honorary doctoral degrees, the Orders of Canada and Ontario, and the Grand Bauhinia Medal and Gold Bauhinia Star, and Justice of the Peace from Hong Kong.
Mini-Exhibition
Organisation: Acquest Stem Cell Research Company Limited
Project Name: Stem Cell Product R&D by Acquest Description: Acquest utilizes research and development to deliver stem cell therapies in a complete solution package to client hospitals across the world for the direct scientific and medical benefit of the patient. Acquest Stem Cell Research’s main functions are as follows: (a) to perform focused and specific research and development in the adult stem cell arena, (b) to provide total solution packages that rapidly enable clinics and hospitals, with limited or no experience in stem cell therapy, to become cutting edge competitors in the marketplace, and (c) the presentation of stem cells in the general marketplace taking advantage of the growing interest for this technology for degenerative diseases.
Contact Person: Mr Pak Liu Email: [email protected] Organisation: Arbele Limited
Project Name: Liver Cancer CAR-T Immunotherapy Description: Cancer immunotherapy as the game changer Chimeric antigen receptor-T (CART) cell based immunotherapy has has attracted a great deal of attention since last years, mainly due to the successful outcome of a number of recent clinical trials in Leukemia patients. In particular, infusion of CART-cell against the antigen CD19 into patients of relapsed and refractory acute lymphoblastic leukemia (one of the most aggressive form of leukemia which fails to response to frontline chemotherapy) resulted in complete remission in upto 90% of the patients. These approaches are therefore suggested as revolutionary cancer therapies that will completely change the paradigm in cancer treatment. Nevertheless, such a success is yet to be replicated in solid cancers. Previous work from our lab has led to the identification of CDH17 as a key protein that promotes the development and progression of hepatocellular carcinoma (HCC). We have subsequently developed an anti-CDH17 monoclonal antibody which not only demonstrated strong inhibitory effects on the growth and metastasis of HCC in vivo, but also significantly sensitized the tumors to the chemotherapeutic compound cisplatin. Building upon these solid and patented findings, we have recently developed the world’s first CDH17-directed CAR-T approach. Taking advantage of this CDH17-CART technology available in our lab, we expect that this new CART therapy will make a major impact in cancer immunotherapy for HCC.
Because of the high incidence and mortality rate, HCC represents a major economic and social burden in Hong Kong. We therefore anticipated that the outcome of our study will produce an immediate economic and social impact on both Hong Kong Government and the general public.
Contact Person: Mr Patrick Ling Email: [email protected] Organisation: Eureka Biotechnology Company Limited
Project Name: PBMC Isolation Machine
Description: The PBMC Isolation Machine is used for isolating the buffy coat from the centrifuged whole blood tube by using machine to improve the recovery rate as well as to reduce the workload of human operators. Contact Person: Mr Ma Mo Email: [email protected] Organisation: HealthBaby Biotech (Hong Kong) Company Limited Description: HealthBaby Group has established for 15 years and committed to providing quality cord blood and umbilical cord banking services with “Only Quality & Fidelity is Worthy of a Lifetime Trust” as the company philosophy. HealthBaby adopted state-of-the-art stem cells storage and processing facilities and is regularly inspected by various international accreditation organizations which include AABB (American Association of Blood Banks), CAP (The College of American Pathologists) and HOKLAS (The Hong Kong Laboratory Accreditation Scheme) to ensure the long-term storage quality of each sample.
HealthBaby devotes abundant resources in biotechnology research projects to explore stem cells medical applications for the betterment of human health. HealthBaby is the first company worldwide to offer cord storage services1. Our exclusive cord technology has already attained Hong Kong, U.S., Taiwan and China patent and we are the only local cord blood bank to have such achievement. The patented technology exclusively extracts the whole cord tissue with unique processing, storage and culture methodology, which can fully enhance the quantity and viability of tissue cells after thawing to help patients in need.
The technology involves mincing the umbilical cord into tissue pieces, each size no larger than
2mm, and admixing them with protein and cryoprotectant to form a mixture, as well as shaking the mixture with special techniques to ensure the stem cells inside are fully protected. Experiments showed that the cord tissues processed by HealthBaby patented technology can successfully be cultured and differentiated into other tissue cells (e.g. adipocytes, chondrocytes and vascular endothelial cells) and the cell colony number derived was over 20 fold of that derived from the non-minced ones.
Besides, HealthBaby proactively co-operated with different academic and medical organizations in R&D projects, which included HIV Vaccine with The University of Kansas Medical Center, HPV Vaccine (undergoing FDA phase 2 clinical trial), as well as the therapeutic effect of human umbilical cord mesenchymal stem cells on acute lung injury caused by severe influenza H5N1 virus infection with local university and Innovation and Technology Commission etc.
HealthBaby introduced automated BioArchive® System, which is specifically designed for stem cells storage and cleared by U.S. Food and Drug Administration (FDA). BioArchive® System adopts closed system design and liquid nitrogen (-196°C ) as storage medium for long term cryopreservation to maintain the quality and viability of stem cells. Report revealed the survival rate of patients who received stem cells for transplant from BioArchive® System was 10% higher than those from traditional tanks2. In Hong Kong, only HealthBaby and public blood bank have used BioArchive® System.
At present, HealthBaby is recommended by most O&G doctors3 and also entrusted by most parents for 8 consecutive years4 in Hong Kong. Our assurance of quality is praised by professionals and clients.
Source: 1. Introduction to the Cord Tissue Market and Cord Tissue-Derived Mesenchymal Stem Cells (CT-MSCs)", BIOINFORMANT, Jun 2015 2. Research result of "National Cord Blood Program" in March 2007 from New York Blood Center 3. IMS 2010 Cord Blood Bank Market Research in Hong Kong (with Private O&G physicians) 4. Ipsos Healthcare 2009-2016 Cord Blood Bank Survey 生寶集團(生寶)成立 15 周年，一直以「唯有誠信與品質，才值得終身信賴」為宗旨，致力提
供最專業優質的臍帶血及臍帶儲存服務。生寶高度重視品質，堅持採用全球最頂尖的幹細胞
儲存及處理系統，並定期接受不同國際認證機構的審查，例如美國血庫協會(AABB)，美國病
理學會(CAP)及香港實驗所認可計劃(HOKLAS)等，以確保樣本長期處於最佳質量，給予客戶最
大的保障。 生寶投放大量資源在研發項目上，不斷開發幹細胞醫療用途，提升人類健康質素。生寶是全
球首家公司推出臍帶儲存服務 1，獨家研發的臍帶處理、儲存及培養技術，已成功取得香港特
別行政區政府知識產權署、美國專利商標註冊處、台灣經濟部智慧財產局以及中國國家知識
產權局認可，成為全港唯一得到四地專利認可的臍帶血庫。此專利涵蓋完整臍帶組織，可全
面促進解凍組織內細胞數量及活性，幫助有需要的患者進行移植。 生寶臍帶專利技術是先將臍帶組織切碎成不多於 2 立方毫米的小塊，注入特別配方的蛋白質
及抗凍保護劑，並以專業技巧將臍帶小塊與抗凍保護劑充份混和，確保組織內的細胞得到充
份保護。實驗研究指出利用生寶專利技術處理的臍帶組織，在解凍後可成功培植及誘導成其
他組織細胞(如脂肪、軟骨、血管內皮等)；在細胞培植數量上更較其他沒有切碎臍帶組織的方
法高出逾 20 倍。 此外，生寶亦積極聯同不同的合作單位研發各種項目，包括與美國堪薩斯大學合作研發愛滋
病疫苗；子宮頸癌融合蛋白疫苗通過美國食品及藥物管理局（FDA）核准，目前已進行第二期
臨床實驗；並與本地大學及創新科技署共同研發臍帶間質幹細胞對治療 H5N1 流感病毒所引起
的急性肺損傷等。
幹細胞儲存方面，生寶使用的 BioArchive®零感染液態氮全自動系統，是專門為儲存幹細胞而
設計，並已通過美國食品及藥物管理局(FDA)認可。BioArchive®系統採用無蓋密閉式設計配合
恒溫液態氮(-196°C)作儲存媒介，可長久維持細胞活最佳活性，保證細胞質量。研究顯示，利
用 BioArchive®系統的幹細胞樣本作移植，病人手術後的存活率比使用傳統開蓋式儲存槽高出
10%2。全港只有生寶及公營臍帶血庫使用 BioArchive®系統。 至今生寶已成為全港最多婦產科醫生推薦 3 的臍帶血庫，同時連續 8 年獲最多父母選擇 4，證
明生寶一直對品質的重視和堅持獲得專業人士和客戶的肯定。
Contact Person: Ms Cindy Wong Email: [email protected] Organisation: Karolinska Institutet, Ming Wai Lau Centre for Reparative Medicine
Project Name: Introduction of Karolinska Institutet, Ming Wai Lau Centre for Reparative Medicine
Description: Karolinska Institutet (KI) is one of the world’s leading medical universities. Our mission is to contribute to the improvement of human health through research and education. KI accounts for over 40 percent of the medical academic research conducted in Sweden and offers the country’s broadest range of education in medicine and health sciences. Since 1901 the Nobel Assembly at KI has selected the Nobel laureates in Physiology or Medicine. KI inaugurated its first overseas branch, the Ming Wai Lau Centre for Reparative Medicine (MWLC), Hong Kong, in October 2016. MWLC operates two nodes, one based in Stockholm, Sweden and the other in Science Park, Hong Kong, with a research laboratory of around 10,000 square feet. The Hong Kong node will be a hub to explore novel technologies in stem cell biology and regenerative medicine, in areas such as biomedical engineering, gene-editing, RNA technology, next-generation single-cell analysis/bioinformatics for the advanced development and translation of specialty areas of stem cell-based regeneration of the heart, liver and nervous systems.
Contact Person: Ms Emily Ip Email: [email protected]
Organisation: Living Tissues Company Limited Project Name: We grow your cartilage Description: Our focus is to repair cartilage damages caused by trauma or degeneration. We use stem cells of the donor to grow tissues of cartilage, bone with the bone/cartilage interface as one construct. The construct in the form of an implantable plug is then implanted into the patient to replace the damaged tissues. We are a spin off from the Tissue Engineering Laboratory at the University of Hong Kong. The Tissue Engineering Lab has developed many technology platforms in the last decade, and these formed the solid foundation of Living Tissues. Contact Person: Mr Sunny Cheng Email: [email protected] Organisation: Novoheart Limited Project Name: Stem cell technology with a heart for innovation Description: The human body is composed of countless cells, but terminally differentiated cells such as those from the heart and brain cannot be regenerated once they are lost or damaged; 'pluripotent' stem cells (PSCs) are a unique stem cell type that can divide perpetually while having the ability to become any tissue type in the body. The world-class scientific team at Novoheart, led by co-founders Professors Ronald Li, Kevin Costa and Michelle Khine, has successfully produced large quantities of ventricular cardiomyocytes (vCMs) from human PSCs, and using these vCMs, they assembled the world's first thumb-sized human "heart-in-a-jar". This paves the way to the production of "heart bandages" for patients after heart attacks, whilst also providing an unprecedented screening platform to revolutionize the discovery of new drugs and therapeutics. Who We Are Dedicated to the application of stem cell technology on medicine and pharmaceutical development, Novoheart is a global biotechnology company founded in Hong Kong, with Innovation Centers being set up in the U.S., and began operations in the Hong Kong Science Park in August 2014. Co-founder Professor Ronald Li, a world-renowned expert in stem cell and regenerative medicine, and his team have successfully produced the world's first human "heart-in-a-jar" using PSCs. What We Do The team of scientists at Novoheart has devoted their efforts to stem cell research, and dramatically improved the production yield of heart muscle cells: from a yield of only 2-3 vCMs per 1,000 PSCs used, to the remarkable 70-80 vCMs yielded from just a single PSC, thus overcoming a significant technological bottleneck. Combined with cutting edge, patented bio-engineering technology, Novoheart has developed for different purposes a series of engineered heart tissue constructs, collectively known as the MyHeartTM Platform, including the first, unique "heart-in-a-jar" in the world.
Why It Matters Novoheart is working with pharmaceutical companies, using the MyHeartTM Platform, to provide highly efficient, multi-faceted pre-clinical safety assessments of hundreds of thousands of drugs and conditions yet with higher fidelity than regular animal testing which regularly fails to predict human heart responses to drugs. This translates into significantly reduced risks to patients, shorter screening times, and drastic reduction in timescale and cost, and ultimately better clinical and commercial successes. The technology used in constructing the MyHeartTM platform could be adapted for the production of "heart bandages" for the repair of damaged heart tissue, potentially providing a solution to the perennial problems of donor shortage and immune rejection associated with transplants, and bringing cures to previously incurable heart diseases. Company website: http://www.novoheart.com 萬能幹細胞複製心臟造福病人 人體由無數細胞組成，然而不少成人體內細胞如心臟或神經細胞都缺乏「再生」能力。「萬
能幹細胞」乃一種特別的幹細胞，可不斷繁衍之餘亦能變成身體任何組織的細胞。再心生物
科技有限公司 Novoheart 創辦人李登偉教授、Kevin Costa 教授及 Michelle Khine 教授領導的科
研團隊利用「萬能幹細胞」製造大量心臟肌肉細胞，成功研發全球首個拇指般大小的迷你人
類心臟，為研製供心臟病治療使用的「心臟膠布」打好基礎，期望最終能解決器官捐贈不足
的問題。迷你心臟更提供了可靠的試藥平台，為研製新藥帶來革命性影響。 研發團隊 再心生物科技是一家於香港創辦的全球生物科技公司，現正在美國設置創新中心。公司於
2014 年 8 月正式加入科學園，專注將幹細胞研究應用於醫學及藥物研發。公司創辦人李登偉
教授是全球知名的幹細胞及再生醫學專家，他和團隊利用萬能幹細胞科技，成功研製成全球
首個人類「迷你心臟」。 研發說明 再心生物科技的科研團隊致力幹細胞的研發，成功大幅提升心臟細胞的製造量：十年前 1,000個「萬能幹細胞」只能製造出 2 至 3 個心室心肌細胞，現在只需一個「萬能幹細胞」，便可
製 70 至 80 個心室心肌細胞，打破業界多年來的技術僵局。加上已註冊專利的頂尖生物工程
技術，再心生物科技已成功研發適合不同應用的一系列心肌組織構造，統稱 MyHeartTM 平台，當中包括全球首個及獨有的「迷你心臟」。 應用及影響 再心生物科技正與藥廠合作，利用「迷你心臟」及有關平台，提供一套臨床前的高效率、全
方位安全評估，能快速測試數以十萬的藥物，比動物測試更能模擬成人心臟對藥物的反應，
有望大大減低新藥物對病人的傷害，亦可革命性地縮短試藥時間，為藥物研發減低成本並提
升成功率。而製造「迷你心臟」的有關技術亦可用於設計修補受損心臟的「心臟膠布」，解
決器官捐贈不足的問題，並減低病人於器官移植手術後身體出現排斥的常見風險，為至今難
以根治的心臟疾病提供治療。
公司網頁: http://www.novoheart.com
Contact Person: Dr Gabriel Wong Email: [email protected] Organisation: OPER Technology Limited Project Name: Autologous Neural Stem Cell (ANSc) Harvest Description: OPER Technology Limited is the first and the only group in the world that is able to safely and specifically harvest neural stem cells from the brain of ive subjects without the risk of life. This trailblazing technology, Autologous Neural Stem Cell (ANSc) Harvest, is already granted US and HK patents and has its patent application filed in China and the EU. ANSc Harvest is facilitated with our strategically designed nanoparticles which not only can automatically and precisely attach on neural stem cells in the brain, also can naturally metabolize in biological processes. This minimally invasive micro-surgery could be real-time monitored by current bio-imaging devices, for example magnetic resonance imaging (MRI). The extracted neural stem cells are multipotent. They can develop into different neural cells in-vitro. Afterwards, the neural cells could be re-injected into the brain of the same live subjects to replace the already degenerated neural cells. ANSc Harvest is going to bring revolutionary changes in research and clinical practice fields. Since this innovative technology does not sacrifice life for stem cells, it allows repeated neural stem cell extraction from the same live subjects, and most importantly, autologous cell replacement therapy for human in the future. Researchwise, ANSc Harvest benefits higher accuracy and consistency in researches related to stem cells, drug discovery and drug development. Repeated extraction of ANScs from the same animals allows researchers to trace the genetic background of the donors and better their research results. It minimizes the sacrifice of laboratory animals too. In clinical aspects, ANSc Harvest and the consequential autologous cell replacement therapy will hugely impact on medical treatments for incurable diseases, including neurodegenerative and cancer diseases. Citing the potential treatment for neurodegenerative diseases as example, patients could use their own neural stem cells to develop healthy neural cells to replace their
already degenerated neural cells, by means of ANSc Harvest, without the risks of immune rejection, ethical issues, tumor formation, brain surgery, genetic instability, etc. The uniqueness and potential applications of ANSc Harvest gains public-wide recognition and won many local and international industrial awards, including: 1) Innovation and Technology Commission ‘Technology Start-up Support Scheme for University’ from Hong Kong Baptist University (2014-2015), (2015-2016) &(2016-2017) 2) The 2nd Taiwan International Congress of Parkinson’s Disease and Movement Disorders – International Travel Award (2015) 3) Red Herring Top 100 Global (2015) 4) Global Entrepreneurship Week 50 (GEW50) Top 50 Startup in the world (2015) 5) The 5th Bank of China (HK) FITMI ‘Technology Start-up’ Award – Gold Award (2015) 6) Hong Kong Awards for Industries: Technological Achievement – Certificate of Merit (2015) 7) GSK Neuro 2020 – Finalist (2016) 8) The 7th Annual China Healthcare Investment Conference Business Plan Competition – Second Place (2016) 9) The 44th International Exhibition of Inventions of Geneva – Gold Medal in Surgery Category (2016) 10) HKBU Innovationem Award (2016) 11) Asia Pacific Entrepreneurial Awards 2016 Hong Kong (Healthcare & Pharmaceutical Industry (2016) 11) Mediazone 2017 Hong Kong Most Valuable Companies (2016) 12) The 5th China innovation and entrepreneurship education [Hong Kong, Taiwan, Macua]- First running-up (2016) 薈新科技有限公司是全球第一及唯一的團隊能夠從活體的腦內安全和特定地抽取神經幹細
胞，並且不威脅該活體的性命。這項創新技術，名為「自體神經幹細胞(ANSc)分離技術」,已獲頒美國和香港專利，其中國及歐洲專利申請也正在審批。 我們策略性研製了一種磁性納米粒子配合 ANSc 分離技術。它們能在腦內自動和準確地找到及
依附著神經幹細胞；此外，它們更有可生物代謝的特性。這項微創手術能配合現有的生物顯
像儀器，如核磁共振成像(MRI)，讓操作者實時監控手術過程。 ANSc 分離技術提取出的神經幹細胞是多潛能性幹細胞，能在體外培養成不同種類的神經細
胞。這些神經細胞能被注入於同一活體的腦內，以取代已退化的神經細胞。 ANSc 分離技術將在科研及臨床醫學層面帶來革新改良。這項全球領先科技不需犧牲生命以換
取幹細胞，因此可以在從同一活體腦內重複抽取神經幹細胞，更有望在將來為人類提供自體
細胞替代療法。 在科研角度出發，ANSc 分離技術有助提高實驗結果的準確性和統一性，有利與神經幹細胞相
關的研究，並能促進研究和開發新藥物治療，加強藥物發展。而重複在從同一活體抽取神經
幹細胞，亦有助科研人員追溯該活體的基因背景，對它們的科研工作帶來莫大好處；同時亦
減少實驗動物的犧牲。 就臨床用途而言，ANSc 分離技術及其引伸的自體細胞替代療法將對不治之症（如神經退化性
疾病和癌症）帶來深遠影響。如以神經退化性疾病的潛在治療為例，在 ANSc 分離技術的幫助
下，病人將能使用他們自己的神經幹細胞來培植他們專屬的神經細胞，以取代他們已退化的
神經細胞；同時大大減低了自我免疫系統排斥、道德爭議、腫瘤產生、腦手術風險、基因不
穩定性等。 ANSc 分離技術的創新性、獨特性和潛在應用性得到廣大認同，亦為薈新科技有限公司贏得不
少本地和國際業界獎項，包括： 1) 香港創新科技署「大學科技初創企業資助計劃」香港浸會大學得獎者 (2014-2015), (2015-2016) & (2016-2017) 2) 第二屆台灣帕金森症與運動障礙疾病國際研討會 – 海外交流獎助 (2015) 3) Red Herring 全球首 100 間初創企業獎 (2015) 4) Global Entrepreneurship Week 50 (GEW50) 全球首 50 間初創企業獎 (2015) 5) 第五屆中國銀行（香港）FITMI 「初創科技」潛能大獎 – 金獎 (2015) 6) 香港工商業獎: 科技成就 – 優異證書 (2015) 7) GSK Neuro 2020 決賽得獎公司 (2016) 8) 第七屆啟珂健康投資論壇商業計劃書大賽 – 第二名
9) 第四十四屆瑞士日内瓦國際發明展－評審團嘉許金獎（手術類别）(2016)
10) 浸大創新獎 (The HKBU Innovationem Award) (2016) 11) 亞太企業精神獎 2016 香港（保健和製藥行業）(2016) 12) Mediazone 2017 香港最有價值公司(2016)
13) 中國創新創業賽港澳台賽 – 第二名 (2016)
