2018-11-03 The 21st SAPA-NE Scientific Symposium

Updated: Apr 9, 2019


21st SAPA-NE Scientific Symposium Report

Huijuan Li, Lin Lin, James Cao


November 3rd, 2018

Richard E Griffin Academic Center

MCPHS University

670 Huntington Avenue Boston, MA 02115


Executive Summary


November 3rd 2018, Boston, the world’s renowned center for education and innovation in the global medical field, welcomed Sino-American Pharmaceutical Professionals Association (SAPA)’s New England Chapter (SAPA-NE) members, pharmaceutical giants, academic elites, world-renowned leaders and researchers to join a full day 21st SAPA-NE scientific symposium at the Massachusetts College of Pharmacy and Health Sciences University (MCPHS). More than 250 SAPA-NE members and friends gathered at the Richard E Griffin Academic Center Auditorium to listen to 12 invited speakers, representing academic institutions, healthcare providers, industry leaders and physician networks.

The conference began with opening remarks and warm welcome by Dr. Jun Johnny Yang, SAPA-NE president; Chairs of the symposium, Dr. Lin Lin and Dr. James Cao; and host of the symposium, Dean of the School of Pharmacy, MCPHS University, Dr. Paul Difrancesco.

Although the journey from concept to clinical success has been long, gene and cell therapy are beginning to bring new treatment options to multiple fields. Recent approvals in both gene and cell therapy, including Kymriah from Novartis; Yescarta from Kite, a Gilead Company; and Luxturna from Spark Therapeutics, and many entering clinical development, have certainly paved the way for the next decade possibly be the new era of evolving therapeutic treatment from phenotypic to genotypic interventions.

Exciting speeches delivered by 12 keynote speakers, including Dr. Khalid Shah, vice chair of Research at Brigham and Women's Hospital (BWH), director of the Neurosurgical Stem Cell Therapy and Imaging Center (CSTI); Dr. Daniel Bauer, Professor, Boston Children's Hospital, Dana-Farber Cancer Institute, Harvard Medical School; Dr. Tony Ho, EVP of CRISPR Therapeutics; Dr. Srinivas S. Rao, Global Head of Translation in vivo Model, Sanofi; Dr. Zdravka Medarova, Associate Professor, MGH, Harvard Medical School; Dr. Jeffrey Lasker, MORE Health Chief Physician Officer; Dr. Jay Hou, VP of Vector Engineering, Voyager Therapeutics; Dr. Alison McVie-Wylie, Ph.D., Head of Gene Therapy, Rare Disease Research, Sanofi; Dr. Zhenhua Wu, CEO of NeuExcell Therapeutics; Dr. Jian Chen, Founder & CEO, Celetrix LLC; Dr. Jing-Tao Wu, VP DMPK and Dr. Amy Zhang, Sr. Director Clinical Pharmacology, Alnylam, centered around the challenges and promises with presentations and interactive dialogues on “The Next Phase of Gene and Cell Therapy”. The topics covered a broad scope from early discovery, development to regulatory approval.

“The success of the symposium would not be possible without the support of event host MCPHS University, organizing committee members’ tireless effort, many volunteers’ commitment, and vendors and sponsors’ support”, summarized Dr. Jun Johnny Yang. “Appreciation goes to everyone who made this symposium a success, especially to our Chairs of the symposium, Dr. Lin Lin and Dr. James Cao for organizing such a quality event with all the speakers as leaders in the field, and our venue coordinator Dr. Hongwei Zhang, Assistant Professor and Director of Pharmaceutical Sciences at MCPHS University”, Dr. Yang continued.

The excitement and discussion continued through the networking session and dinner table. “I enjoyed the event and was really impressed by the quality of the science and the participation from your association”, commented by Dr. Alison J. McVie-Wylie. “It was a well-organized scientific symposium and we were happy to be part of it”, shared by Dr. Jay Hou and Dr. Jing-tao Wu. Many of us have and will continue to witness how innovation and technology have been revolutionizing pharmaceutical industry and our everyday work.

SAPA- NE continues our mission to inspire scientific discussion and exchange, foster collaboration and dialogues among discovery research, development and regulatory to ultimately deliver to patients of safe and cost-effective treatment for unmet medical needs. This dialogue will continue as the SAPA-NE 2019 annual conference theme “Developing and Delivering the Advanced Medicines to All Patients”, announced by our president-elect Dr. Qiying Hu. On behalf of the organizing committee, we all look forward to seeing you at the 21st SAPA-NE Annual Conference, Boston Marriott Cambridge, MA, USA on June 8th, 2019!


21st SAPA-NE 科学研讨会---波城秋日一抹绚丽的学术风景线

        2018年11月3日, 在这姹紫嫣红的金秋时节,全球医药领域教育、研发、创新的圣地---波士顿迎来了新英格兰美中医药开发协会(SAPA-NE)有史以来最为火热的第21届科学研讨会, SAPA-NE会员、医药界大牛、学界精英、世界名企领袖和广大科研人员等专业人士欢聚在位于波士顿哈佛医学中心地带的麻州药学院Richard E Griffin 学术中心礼堂 ,聆听来自哈佛医学院等高等学府、全球知名药企、科技创新新锐的12位知名科学家及药物研发领袖的精彩演讲。会议围绕着“基因和细胞疗法的未来”这个主题,对话了这一领域的突破性进展、面临的挑战机遇及未来的发展愿景等。内容丰富多彩,涵盖了从药物的早期研发、后期开发和监管审批等各个环节。在基因和细胞治疗日渐火热的时下,本次研讨会创历史记录地吸引了250多位来自波士顿地区各大药企、初创公司、著名高校等高层次科研人员参会,人满爆棚、异常火热、所有入场票全部售罄。会议演讲精彩、讨论热烈、所有内容结束后人们仍余兴未了、久久不愿离去,成为波士顿秋日的一抹绚丽学术风景线。

研讨会在SAPA-NE现任会长Jun Johhny Yang(杨军)博士、 科学研讨会主席Lin Lin(林琳)博士和James Cao(曹建明)博士、东道主MCPHS大学药学院院长Paul Difrancesco博士的热情洋溢的欢迎下揭开帷幕。

来自哈佛医学院附属布列根妇女医院(Brigham and Women's Hospital, BWH)科研副院长,并兼任神经外科干细胞治疗和成像中心(CSTI)主任、哈佛干细胞研究所研究员等数职的 Khalid Shah博士,来自儿科排名世界第一的著名的哈佛医学院附属波士顿儿童医院(Boston Children’s Hospital)及世界闻名的癌症专科医院(Dana-Farber Cancer Institute)的Daniel Bauer博士,享誉全球的哈佛医学院附属马萨诸塞州总医院的Zdravka Medarova博士,具有从前期研发到后期申报等一系列丰富经验的现任CRPSPR Therapeutics执行副总裁Tony Ho博士,  来自世界著名大型制药企业Sanofi(赛诺菲)的高级主管Srinivas S. Rao博士及罕见病基因治疗及药理学研究的负责人Alison J. McVie-Wylie博士,来自以AAV基因治疗见长的Voyager Therapeutics的副总裁Jay Hou博士,来自致力于创新性神经系统药物研发的 NeuExcell Therapeutics的首席执行官Zhenhua Wu (吴振华) 博士,来自于MORE Health (爱医传递)首席执行官Jeffrey Lasker博士,来自于专业开发细胞转染技术的公司Celetrix的首席执行官Jian Chen (陈健)博士 ,来自著名药企Alnylam的掌管毒理研究的副总裁Jingtao Wu (吴惊涛)博士与临床药理总监Amy Zhang博士等12位讲演嘉宾分别围绕基因和细胞治疗进行了主题演讲。从肿瘤干细胞治疗到第一个被FDA批准上市的RNAi药物Onpattro诞生,嘉宾的演讲各个精彩,高潮迭起。

“本次研讨会得到了MCPHS大学的大力支持,研讨会的成功是和组织委员会成员的不懈努力,许多志愿者的无私奉献,以及供应商和赞助商的支持分不开的”,Jun Johnny Yang博士总结道。 “感谢所有使本次研讨会取得成功的人,尤其是我们的研讨会主席Lin Lin博士和James Cao博士, 我们的场地协调员Hongwei Zhang博士,MCPHS大学助理教授和药学科学主任,“杨博士继续说道。

会议尾声,SAPA-NE继任会长Qiying Hu(胡琪颖)博士进行了总结发言,宣布SAPA-NE 将于2019年6月8在位于查尔斯河畔的Boston Marriott Cambridge, MA, USA 举行主题为“为所有患者开发创新药物”的第21届SAPA-NE年会,热烈欢迎广大医药界朋友光临2019 SAPA-NE年会,期待在年会的学术大餐上重聚首、再相逢!





Annual scientific symposium is one of Sino-American Pharmaceutical Professionals Association (SAPA)’s New England Chapter (SAPA-NE)’s traditions. Relevant and exciting topics are selected and academic and industry leaders are invited to share and discuss the most advanced progress in the selected fields. Given the rapid proceedings made in the gene and cell therapy in the last few years, it is due for us to repeat this topic though it was covered in 2015 (18th SAPA-NE SS).

A full day 21st SAPA-NE scientific symposium, on Nov 3rd 2018, brought together more than 250 members and friends of SAPA-NE including industry leaders, scientists, academic researchers, and physicians.

The conference began with opening remarks and warm welcome by Dr. Jun Johnny Yang, SAPA-NE president; Chairs of the symposium, Dr. Lin Lin and Dr. James Cao; and host of the symposium, Dean of the School of Pharmacy, MCPHS University, Dr. Paul Difrancesco.

The central dogma of Molecular Biology, the transfer of information from gene sequence to a protein product, has been part of the biotechnology medicine revolution in the past 40 years. Over 400 recombinant proteins including antibodies have been approved which revolutionized the industry to become biological based in the last 10 years as illustrated in the Figure below.




Saltzman E and Rice M, 2018, Advanced Therapeutic Webinar, Defined Health, a Cello Health business.



Although the journey from concept to clinical success has been long, gene and cell therapy are beginning to bring new treatment options to multiple fields. A historical overview of HSC and AAV gene therapy, and CAR-T cell therapy is outlined as the following (Dunbar CE et al, 2018, Collins FS and Gottlieb S, 2018).




Dunbar CE et al, 2018, Gene therapy comes of age. Science.12; 359(6372).

Recent approvals in both gene and cell therapy, including Kymriah from Novartis; Yescarta from Kite, a Gilead Company; and Luxturna from Spark Therapeutics, and many entering clinical development, have certainly paved the way for the next decade possibly be the new era of evolving therapeutic treatment from phenotypic to genotypic interventions (illustrated in the table below).






Saltzman E and Rice M, 2018, Advanced Therapeutic Webinar, Defined Health, a Cello Health business.

The invited speakers, representing academic institutions, healthcare providers, industry and physician networks, discussed the challenges and promises with presentations and interactive dialogues on “The Next Phase of Gene and Cell Therapy”. The topics covered a broad scope from early discovery, development to regulatory approval.

The morning session started with keynote Speaker Dr. Khalid Shah, vice chair of Research at Brigham and Women's Hospital (BWH), director of the Neurosurgical Stem Cell Therapy and Imaging Center (CSTI). Dr. Shah provided an assessment for the current state of cancer stem cell therapy and shared the rationale design for translating the most promising preclinical studies into the clinic. Dr. Shah’s laboratory has shown that different stem cell types home to sites of cerebral pathology and thus can be armed with therapeutic transgenes, a strategy that can be used to inhibit tumor growth by targeting angiogenesis or selectively inducing apoptosis in proliferating tumor cells in the brain. They have engineered different stem cells types to release therapeutic proteins to specifically induce apoptosis in tumor cells and anti-angiogenic to inhibit tumor angiogenesis and tested them in primary and metastatic mouse tumor models. In recent years his laboratory has also focused on targeting tumors that are resistant to different therapeutic drugs and oncolytic viruses.

Dr. Shah shared insights how CRISPR (clustered regularly interspaced short palindromic repeats) technology can potentially engineer cancer, NK and T- cells for the continued advancement in the personalized treatment by introducing a “safety switch” and “bifunctional receptor”; differentiating “Hot and Cold tumors” and “Resistant and Sensitive Patients”. Dr. Shah concluded his talk by summarizing the following: 1) developing tumor models that mimic the clinical setting of tumor growth and pathology is necessary for developing novel therapeutics for cancer; 2) engineering bimodal therapeutics and incorporation of mechanism based therapies that allow targeting multiple pathways is important to develop next generation of cell base therapies for cancer; 3) understanding the current treatment regimen for each cancer type is necessary for translation of novel therapies into clinical settings.