Youth Talks 第七十八期（中文）| 生物医疗领域的创业探索

北京时间10月22日晚八点，iCANX Youth Talks第七十八期将隆重开播！本期，我们邀请到了Asklepios BioPharmaceutical, Inc非病毒递送部门的创始总监高正宏、深圳市中科先见医疗科技有限公司创始人吴天准主讲，浙江大学博士生导师刘帅、路易斯安那州立大学助理教授蔡琪担任研讨嘉宾，电子科技大学张翼教授担任主持人，期待你一起加入这场知识盛宴。

This presentation will elucidate the challenges, complexities and recent advancements pertaining to the delivery of therapeutic molecules to extrahepatic tissues facilitated by novel nanoscale drug delivery system (nanoDDS). NanoDDSs have emerged as mainstream nanoscale carriers for a diverse array of therapeutic cargoes, including peptides, nucleic acids, and gene-editing tools, ushering in novel therapeutic paradigms. However, while these systems have demonstrated efficacy in hepatic tissue, extending their delivery capabilities to extrahepatic targets presents formidable challenges, particularly in tissues characterized by intricate biological barriers such as the central nervous system.

To address this challenge, integrated strategies are being devised, encompassing fine-tuning tissue specific structure-property relationship of nanoparticle, local-environment responsive release mechanisms, advanced administration devices used in clinic practice, and receptor-mediated targeting delivery systems. These approaches aim to achieve precise and efficient delivery of therapeutic cargoes to challenging tissues to achieve therapeutic benefit, while minimizing off-target effects and systemic distribution.

While several strategies have been explored in early-stage clinical trials to advance viral vector-based gene therapy in extrahepatic tissues, the full potential of nanoscale delivery remains to be realized for expanding the landscape of new therapeutic modalities. Recent preclinical investigations have showcased targeted nanoparticles delivery of versatile therapeutic molecules to central nervous system in disease relevant animal models, highlighting the potential for transformative therapeutic development in a wide range of diseases. 

With a special focus on CNS, this presentation will cover 1) remotely controlled nanotechnology for local modulation of blood brain barriers to facilitate the delivery of biotherapeutics; 2) super-resolution method coupled with single nanoparticle tracking for unveiling the drug diffusion barrier in the CNS-extracellular space (ECS) and its physiological properties, and impact on the drug delivery. These advancements represent significant strides in the field of nanotechnology-mediated extrahepatic tissue delivery and offer promising avenues for future therapeutic innovation and clinical translation.

Dr. Gao is a creative scientist specializing in innovative nanoscale delivery technology for enabling new drug discovery and development. With over ten years of experience at leading academic research institutions and biotech companies, he is actively engaged in advancing biomedical research, developing new technologies, and translating them into medical applications. His extensive research portfolio includes nanomedicine, molecular imaging, liquid biopsy, medical devices, gene delivery, nanoparticle diffusion, and gene therapy and genomic editing. Over the years, Dr. Gao's has pioneered several nanoscale delivery technologies to understand and overcome biological barriers in the central nervous system, including brain-extracellular space (Nature Nanotech.; 2017; Nanomaterials 2017; Polym J., 2018; ACS Nano 2017) and blood-spinal cord barrier (BioRxiv., 2022; ACS Nano 2024, submitted), for improved drug and gene delivery (Nanoscale Adv., 2024); he has also developed integrated molecular targeting techniques for the early detection of cancers (Sci. Rep., 2017) and conducted "First-in-human" clinical trials (Gastroenterology 2017).

Currently, Dr. Gao serves as the Director and Head of Non-Viral Delivery Department at Asklepios BioPharmaceutical, Inc., a flagship gene therapy company under Bayer. In this role, he leads a team dedicated to advancing non-viral vector-based drug delivery and novel nucleic acid for enabling new generation of life-saving medicine, such as therapeutic gene augmentation and genomic editing. Previously, Dr. Gao was an Assistant Professor at University of Texas at Dallas, with affiliation to UT Southwestern Medical Center. Dr. Gao has received several prestigious awards, including the DOD Therapeutic Idea Award for ALS treatment. He has published over 40 articles in journals such as Nature Nanotechnology, Journal of the American Chemical Society, and Gastroenterology, etc, and is a sought-after keynote speaker. He holds several US patents, and his research has been featured in Science and Nature. 

Chip technologies based on semi-conductor such as microfluidics, bioMEMS and so on have achieved rapid progress and broader applications. The precision, mass production and powerful integration capacity of semi-conductor technology have endowed a fully new perspective and system reconfiguration ability for precision diagnostics. This talk will share our innovative researches and industrial practices in Shenzhen, China in the recent 10 years. We have explored the “Chip+Medicine” technology platform towards the commercialization in single-molecular ultra-sensitive detection and point-of-care technology (POCT). We will exhibit the highly integration and digitalization potential of the advanced biochips, especially the huge advantages for the population of precision medicine in the field of high-sensitivity in vitro diagnostics (IVD). New concept to realize “A chip is an instrument, as well as a consumable” can be achieved based on the world-first high-efficiency monodisperse particle array and low-cost, high-precision multiple-casting processes, to enable the fast spread of nowadays expensive single-molecule detection to primary health care even finally at-home self-testing. We will briefly introduce how CAS-Envision, the spin-off from Shenzhen Institutes of Advanced Technology, Chinese Academy of Sciences (SIAT CAS), converted the intelligent patents into products and explored the commercialization approaches focusing on “Chip+Medicine”. My personal experiences and opinions on “Scientist Entrepreneurship” will be shared at the end.

Tianzhun Wu obtained the bachelor's and master's degree from the Department of Precision Instruments at Tsinghua University, and the Ph.D. from the University of Tokyo. After that, he served as a postdoctoral researcher at the University of Tokyo and Osaka University, and a lecturer at Sun Yat-sen University. In 2013, he joined the Shenzhen Institutes of Advanced Technology, Chinese Academy of Sciences (SIAT CAS) as an associate professor, where he founded the Center for Micro/Nano Systems and Bionic Medicine. In 2016, he was promoted to full professor and Ph.D. supervisor. In terms of industrialization, in 2018 he founded CAS-Envision Medical Technology Company and in 2021 the subsidiary company Shenzhen Bloom Origin Semiconductor. In 2022 he was granted the financial support funded by the Nanshan government to lead the IVD Innovation Platform, National Technology Innovation Center in Guangdong-Hong Kong-Macao Greater Bay Area. So far his accumulation of R&D grant budget is more than 180 million RMB in the area of semiconductors and medical devices. Dr. Wu has nearly 20 years of R&D experience and industrial practice in semiconductors and biomedicine, with research interests including BioMEMS, MicroTAS, nanomaterials, biomimetic interfaces, and neuroelectronics. He has published more than 60 SCI papers and over 100 EI papers, and holds more than 100 domestic and international invention patents (more than 50 among them have been transferred or industrialized). He has been granted as an outstanding youth of the Guangdong Natural Science Foundation and Caliber B of the Shenzhen Peacock Plan. He has once served as a technical committee member (TPC) of IEEE MEMS, senior member of IEEE, a director of the Chinese Society of Micro and Nano Technology, a executive committee member of the Health Data and Digital Medicine Branch of the CPAM, a member of the Standardization Committee of the Chinese Society of Biomedical Engineering, and so on.