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· Discovery and Development of Nucleic Acid Medicines
· Approved RNA Therapeutics
· Delivery Mechanisms of RNA Therapeutics
· Extra-Hepatic Delivery Approaches
· Salient Features of RNAi Therapeutics
· Future of RNA Medicine: Challenges and Opportunities
I willpresent several modalities whereby synthetic RNA can be used to prevent or treat cancer: mRNA vaccines and RNA therapies
-Synthetic messenger RNA can be used to generate anti-cancer vaccines
-Individualised anti-cancer vaccines can be cumbersome to produce but we found a new solution to address this issue
-Synthetic RNA molecules containing chemotherapeutic residues can also be used for therapy of cancer
Antisense oligonucleotide (ASO) therapeutics offer significant promise for treating a wide range of diseases; however, challenges persist in optimizing efficacy, minimizing off-target effects, and achieving tissue-specific delivery. We present a novel platform that combines custom bridged nucleic acid (BNA) amidite chemistry with peptide ligand conjugation to engineer potent shortmer ASOs with enhanced precision and reduced off-target activity. To further improve therapeutic performance, we employ rational peptide ligand design to selectively enhance tissue-specific uptake while minimizing liver and kidney accumulation. Our approach utilizes the Magic Bullet Designer, an AI/ML-driven platform, to engineer tissue-specific peptide ligands for RNA therapeutics, enabling highly targeted and effective delivery. Applying this integrated strategy, we have developed a next-generation RNA therapeutic for aggressive cancers, demonstrating improved safety, efficacy, and tissue specificity.
RNA interference (RNAi) is an evolutionarily conserved post-tranional gene silencing mechanism triggered by small interfering RNAs (siRNAs). RNAi-based therapeutics have emerged as a promising class of medicines with the capability to suppress the expression of virtually any protein-coding gene. Previously, we reported that single-stranded Ago2-processed interfering RNA (saiRNA) can circumvent the association of mature siRNA with non-nucleolytic Argonaute (Ago) proteins, thereby significantly reducing off-target effects mediated by these proteins. In this study, we designed chemically modified saiRNAs with enhanced stability in both cellular and serum environments, without compromising their on-target silencing efficacy. Furthermore, when administered intravenously in mice, fully modified saiRNAs bearing a GalNAc moiety at the 3’ terminus achieved sustained and efficient knockdown of target gene expression. Notably, these saiRNAs exhibited reduced off-target effects mediated by both the guide and passenger strands, compared to canonical siRNAs targeting the same sequences. Collectively, these findings demonstrate that saiRNA represents a potent and promising alternative to conventional siRNA for RNAi-based therapeutics.
The selective induction of RNAi in target cells using therapeutic siRNA remains a major challenge for cancer therapy. Systemic injection of formulated siRNA drug candidates usually leads to liver overload and side effects. Some delivery approaches aim to transport the payload to specific cells in order to increase the amount of oligonucleotide reaching the cancer target tissue, but still leading to high amounts in non-target tissue.
In contrast to a specific delivery approach to induce cell specific RNAi, we investigated a prodrug strategy: A short peptide was coupled to the sensitive 5`end of a siRNA antisense strand in order to inhibit siRNA function. The peptide contains a cleavage site for a protease with specific activity in cancer target cells, leading to peptide cleavage and cell specific siRNA activation. In non-cancerous cells, however, the peptide-siRNA prodrug remains inactive and is not able to induce RNAi. Proof of concept has been demonstrated in various cancer types, including breast and ovarian cancer in combination with several delivery strategies including LNPs and polymer-based nanoparticles. In vivo after systemic injection of LNP formulated peptide-siRNA, drug compounds were still found in high amounts in non-target tissue, but not leading to gene silencing, whereas in xenotransplant tumors induction of RNAi was observed. In the last months, peptide-siRNA manufacturing process was scaled-up in preparation for GMP manufacturing.
We believe that the peptide-siRNA prodrug strategy can significantly reduce side effects of therapeutic siRNAs in non-target tissues and explore new concepts for siRNA targets and sequence design.
We have developed LuxiAPTM, a proprietary antisense oligonucleotide (ASO) drug discovery platform leveraging unique artificial nucleic acids (XNAs). LuxiAPTM has been proven through animal studies to minimize neurotoxicity and immune response while achieving long duration. Extensive background data from this platform has been utilized in collaborations with multiple global pharma companies and technology licensing, leading to steady progress in drug discovery for neurological disorders and other ex-hepatocyte. We’d like to explain current progress of our ASO science and programs.
Lipid nanoparticles (LNPs) are the cornerstone of non-viral mRNA delivery systems, yet their therapeutic potential remains constrained by inefficient endosomal escape, with cytoplasmic delivery rates as low as 2%. Traditional approaches to enhancing endosomal release have primarily focused on chemical modifications of ionizable lipids. Here, we introduce a novel strategy centered on engineering the internal architecture within core-shell LNPs to substantially improve mRNA delivery efficiency. By utilizing ionizable lipid-coated gold nanoparticles (AuNPs) as a demonstrative core, we systematically optimized the LNP nanostructure, achieving a twofold increase in endosomal escape efficiency and a 100-fold enhancement in mRNA diffusion within the cytoplasm compared to conventional LNPs. This structural optimization also promotes more uniform and stable nanoparticle assembly. In vitro studies demonstrated higher mRNA expression and transfection efficiency in HeLa and 293 cells treated with engineered core-shell LNPs. In vivo evaluations revealed a nearly fivefold increase in mRNA expression in murine models, alongside enhanced immunogenicity in SARS-CoV-2 spike-mRNA vaccines and therapeutic efficacy in a triple-negative breast cancer model. Comprehensive biocompatibility and biosafety assessments confirmed the favorable safety profile of the engineered LNPs. This internal architecture optimization framework provides a scalable and adaptable pathway for advancing mRNA therapeutics and vaccines, addressing a key bottleneck in endosomal release and paving the way for broader biomedical applications.
SML Biopharm presents a novel approach to therapeutic antibody development using its proprietary mRNA-NP platform, enabling invivo expression of monoclonal and bispecific antibodies. This strategy circumvents traditional protein manufacturing by delivering mRNA that encodes functional antibodies directly into the body, where they are rapidly expressed. As a primary case study, we showcase the development of an mRNA-based antibody therapeutic targeting Severe Fever with Thrombocytopenia Syndrome (SFTS), a tick-bomeviral infection with high mortaity and no approved treatment. In animal models, the approach demonstrates high expression levels, target specificity, and proteclive efficacy. Furthermore, the plaform's fiexiblly allows expansion to bispecific antibodies, offering potential applications in oncology, autoimmunity, and infectious disease. This presentation will detail the mRNA construct design, LNP formulation parameters, and in vivo proof-of-concept data supporting this next-generation therapeutic strategy.
1 A Novel mRNA-Based Therapeutic Vaccine Targeting HPV16/18 Oncoproteins: Preclinical Efficacy in Tumor Regression and Immune Activation
Liang Du, Abogen Biosciences
2 ABO1105, A Novel Lyophilized RSV mRNA Vaccine: Design, Immunogenicity and Protection Efficacy
Siyu Tian, Abogen Biosciences
3 Development of Next-Generation Ionizable Lipid for Intravenous mRNA Therapy
Xinpeng Ma, Abogen Biosciences
4 Overcoming Safety Challenges in mRNA Vaccine: Development of Next-Generation Ionizable Lipid
Xinpeng Ma, Abogen Biosciences
5 Efficient Circularization of Protein-Encoding RNAs via a Novel Cis-Splicing System
Shaojun Qi, Abogen Biosciences
6 Systemically delivered lipid nanoparticle-mRNA encoding lysosomal acid β-glucosidase restores the enzyme deficiency in a murine Gaucher disease model
Xingdong Guo, Immorna (Shanghai) Biotechnology, Co. Ltd.
7 Understanding the correlations between pKa and the cytosolic delivery of the mRNA-lipid nanoparticles
Yujia He, Monash University
8 mRNA-Delivered Neutralizing Antibody Provides Complete Protection Against Severe Fever with Thrombocytopenia Syndrome Virus
Jaeyong Kim, SML Biopharm
9 Integrated Off-Target Mitigation in Antisense Therapeutics via the BROTHERS™ Platform and Nucleic Acid Chemistry
Kaho Oh, Liid Pharmaceuticals
10 Engineered circRNA Vaccine Expressing Optimized FHbp-NHBA Fusion Antigen Elicits Robust Cross-Protection Against Epidemic MenB Lineages
Qiqi Zhang, Guangzhou Lab
11 Circular RNA Technology-Driven Tissue Repairing
Zengjin Huang, Hexagon BioMed Lab
12 A phase 1b trial results of PCNAT-01, a personalized neoantigen cancer vaccine in pancreatic ductal adenocarcinoma
Asma Khanniche, Anda Biology Developement Medicine (Beijing) Co.Ltd
RNA-LNP (lipid nanoparticle) vaccines have revolutionized vaccine development, cancer treatment, and gene therapy. However, conventional solvent-driven RNA-LNP production relies on centralized manufacturing and storage infrastructure, inherently limiting personalized assembly in point-of-care. Here, we introduce Rapid On-site Modular Assembly (ROMA), a solvent-free platform enabling point-of-care synthesis of customizable RNA-LNPs with industrial-scale efficiency. ROMA exploits high-frequency oscillatory flow (10^2 to 10^4 Hz) in engineered microfluidic channels to encapsulate diverse RNA payloads (1 to 13 kb) into pre-formed empty LNPs within 1 minute, achieving > 90% encapsulation efficiency at low N/P ratios (2 to 6). The resulting LNPs are structurally and functionally isomorphic to conventional counterparts, matching key benchmarks: size (80 to 200 nm), monodispersity (PDI < 0.1), transfection efficacy (> 97%), and in vivo biodistribution. A fully automated, portable ROMA prototype (< 2 kg) generates 200 ready-to-use doses/hour with a variability of < 5%. In animal studies, six ROMA SARS-CoV-2 vaccine candidates—produced via orthogonal pairing of three RNA types and two LNP formulations—elicit comparable antibody titers and safety profiles to conventional ones. This platform bridges the gap between centralized manufacturing and point-of-need production, democratizing access to real-time, customizable RNA therapies.
To combat antimicrobial resistance (AMR), a leading cause of death worldwide, TheraRNA has established an innovative and highly efficient mRNA vaccine design platform powered by Artificial Intelligence. This platform fully integrates AI-driven antigen screening, structural prediction, sequence optimization, and a proprietary delivery system, enabling the development of several first-in-class mRNA vaccine candidates.
This presentation will showcase preclinical data for our broad-spectrum, cross-species vaccines targeting critical drug-resistant bacteria, including Staphylococcus aureus, Streptococcus, and Enterococcus. We will demonstrate both the preventive and therapeutic efficacy of these candidates in animal models. Furthermore, the talk will introduce the platform's promising applications against other challenging pathogens, such as the malaria parasite (Plasmodium).
1. The promise of in vivo CAR-T as the next generation of cell therapy
2. How LNP technology enables safe and effective in vivo T-cell engineering
3. Leveraging clinical experience with LNP to accelerate development
RNA-based therapeutics have sparked a revolution in modern medicine, with the potential to transform the way we treat diseases. Among these, RNA editing emerges as a particularly promising strategy, allowing for the temporary installation of non-heritable edits. This approach opens new opportunities for treating both genetic diseases and common complex disorders. In this talk, I will present two RNA editing tools developed in our laboratory. The first method, SCISSOR, allows for flexible modification of open reading frames. We demonstrate its potential both in repairing frameshift mutations and in introducing frameshifts to generate immunogenic tumor poly-epitopes. The second method, MIRROR, utilizes engineered oligoribonucleotides to recruit endogenous ADAR enzymes for programmable RNA base editing. Through rational design based on highly edited natural substrates, MIRROR enhances RNA editing efficiency both in vitro and in vivo. Collectively, these innovative tools hold considerable promise for advancing RNA therapy and biomedical research.
Scarless circular mRNA exhibits superior anti-tumor activity by prolonged and enhanced CAR expression ·CellectLNP is a versatile platform for modular engineering of antibody-conjugated LNPs to achieve efficient and selective in vivo CAR cmRNA delivery to T cell · In vivo CAR-T based on cmCAR and CellectLNP provides novel opportunities for autoimmune diseases and cancers
-Translation of circRNAs, mechanism and regulation
-Develop the new technology platform for circRNA drugs
-Brief introduction of several circRNA drug pipelines
Epstein-Barr virus (EBV) is strongly associated with B cell lymphoma and nasopharyngeal carcinoma (NPC). In China alone, there are over 80 thousand new cases of NPCs each year and EBV is present in each of these cases. Thus this virus is the cause of significant morbidity globally. Maintenance of genomic stability is essential for homeostasis and organismal longevity. The most dangerous form of DNA damage is a double-stranded break (DSB). The DNA damage response (DDR) and subsequent repair ensure faithful duplication and inheritance of genetic material. In my presentation at the RNA World 2025 meeting, I will be discussing how EBNA2 promotes immune evasion by activating DDR through alteration of miR-26a and miR-155 expression. Our data suggest a novel mechanism of humoral immune evasion by EBV EBNA2 to inhibit class switch recombination.
Ovarian cancer (OC) is a lethal gynecologic malignancy with limited response to current immunotherapies such as anti-PD-1/PD-L1. We developed a 3D OC model simulating immune-tumor interactions in a physiologically relevant environment to enable preclinical testing of miRNA-based combination therapies. Our results show that miR-200c enhances CD4+ and CD8+ T cell activity, reduces stemness, and impairs tumor growth in a fibrin-based 3D system. A combinatorial nanomedicine approach using gold nanoparticles (AuNPs) functionalized with trastuzumab (TZ) and delivered to miR-200c-overexpressing OC cells was evaluated. This dual strategy significantly reduced cell viability, downregulated HER2 signaling, and promoted apoptosis, highlighting the potential of miRNA-assisted nanotherapies to overcome resistance in HER2-low or heterogeneous ovarian tumors.
Dr. Muthiah (Mano) Manoharan serves as a Senior Vice President and a Distinguished Research Scientist at Alnylam Pharmaceuticals, Cambridge, Massachusetts, USA. Dr. Manoharan joined Alnylam in 2003. He built the chemistry group at Alnylam and pioneered the discovery and development of RNA interference-based human therapeutics. Dr. Manoharan has had a distinguished career as a world-leading chemist in the areas of oligonucleotide chemical modifications, conjugation chemistry, and delivery platforms (lipid nanoparticles, polymer conjugates, and complex-forming strategies). He is an author of more than 200 publications (nearly 37,000 citations with an h-index of 85 and an i10-index of 329) and over 400 abstracts, as well as the inventor of over 225 issued U.S. patents.
Dr. Bo Ying has dedicated over a decade of his career to the technology research into nucleic acid -based therapeutics and the development of transformative drugs. He founded Abogen, a biotechnology company with a mission of curing otherwise untreatable diseases through mRNA science and technology.
As the founder, Dr Ying also has served as Chairman of the board and CEO of Abogen. Under his leadership, the company has established its own proprietary mRNA platform and delivery technology that allowed the development of the therapeutics and vaccines for infectious diseases, cancer, and rare diseases. Most recently, Abogen’s mRNA platform capabilities have come together to allow the authorized use and approval of first China made mRNA vaccine against the COVID-19 pandemic.
Prior to founding Abogen, Dr Ying had held several roles at numerous leading biopharmaceutical and biotechnologies companies in the US, mainly responsible for leading drug formulation, process development, technology transfer and manufacturing. Across his career, he takes pride in a broad array of innovative drug developing experiences in siRNA, mRNA-based therapies for tumor immunotherapy、cancer vaccines and gene-editing drug product candidates.
Dr. Ying received his bachelor’s degree in life sciences from Fudan University, Shanghai, and Ph.D. in pharmaceutical sciences from Northeastern University. He is credited as the inventor of a dozen issued patents and patent applications, and his work appears in journals such as Cell and Lancet.
Over the years, Dr. Ying has held several positions in influential organizations. From 2018 to 2019, he served as the president of the Chinese-American BioMedical Association (CABA) in Boston, where he made contributions to the advancement of science and technological innovation with great potential to improve human health.
Dr. Blakney is an Assistant Professor and Tier 2 Canada Research Chair in the Michael Smith Laboratories and School of Biomedical Engineering at UBC. She received her Bachelor of Science in Chemical & Biological Engineering from the University of Colorado at Boulder, and her PhD in Bioengineering from the University of Washington. She completed a postdoctoral fellowship at Imperial College London on the development of molecular and biomaterial engineering strategies for delivery of self-amplifying RNA. Her lab uses bioengineering, molecular biology and immunology approaches to develop the next generation of RNA vaccines and therapies. Her research has been published in a variety of top tier journals including ACS Nano, Nature Communications, Molecular Therapy, Biomaterials, Journal of Controlled Release, and Advanced Materials. She is also a passionate science communicator and runs a TikTok channel dedicated to educating the public about RNA biotechnology, which now has >250,000 followers and >18M views.
Dr. Blakney has received numerous awards and recognitions in addition to the Tier 2 Canada Research Chair in Nucleic Acid Bioengineering, including the 2023 MIT Tech Review’s 35 Innovators Under 35, 2022 Gairdner Early Career Investigator Award, the 2021 UBC President’s Award for Public Education Through Media and the 2022 Controlled Release Society Gene Delivery and Editing Focus Group Young Investigator Award.
Dr. Zhen Li cofounded ADARx Pharmaceuticals in 2020 and is the President and CEO of ADARx. She has more than two decades of experience in research and development in the pharmaceutical and biotech industries. Prior to ADARx, Dr. Li served as Senior VP at Arrowhead Pharmaceuticals where she led discovery, research and non-clinical development of Arrowhead’s RNA delivery platform and multiple product candidates. Previously, Dr, Li was a director at Merck and led a team dedicated to siRNA development. Dr. Li received her PhD in Chemistry and Chemical Biology from Harvard University and her B.S. from Peking University.
Trained as an immunologist at the Pasteur Institute (Paris, France), Steve Pascolo used mouse models to test and develop mRNA-based vaccines (direct injection of mRNA) during his post-doc in Tuebingen, Germany from 1998 till 2000. In 2000, he co-founded CureVac with Dr. Hoerr and Dr. von der Mulbe. Steve Pascolo was Chief Scientific Officer (CSO) of the company from 2000 till 2006, developing the technology, implementing the worldwide first pharmaceutical (GMP) production of synthetic mRNA and starting in 2003 the worldwide first clinical studies where humans (including first himself) got injections of in vitro transcribed mRNA. In 2006, he joined the oncology department of the University Hospital of Zurich, Switzerland and continued the development of immunotherapies based on RNA. In 2008, he founded Miescher Pharma to support this work. In 2013, he joined the dermatology department of the University Hospital of Zurich, Switzerland. In 2017, Steve Pascolo implemented in the University of Zurich an academic mRNA platform https://www.cancer.uzh.ch/en/Research/mRNA-Platform.html. In collaboration with several research and clinical departments in Zurich he optimizes, tests and implements mRNA based vaccines and therapies.
Paul Panorchan is currently located in Shanghai and serves as President of R&D China at BioNTech, overseeing the development of novel cancer immunomodulators and targeted therapies. He has two decades of experience in the pharmaceutical industry with expertise in early clinical development, drug metabolism and pharmacokinetics, clinical and quantitative pharmacology, and translational medicine. Before joining BioNTech, Paul worked in Boston at Vertex Pharmaceuticals and contributed to several global approvals of novel therapies such as Orkambi and Symkevi. He earned his Ph.D. in chemical and biomolecular engineering from Johns Hopkins University in 2006.
Dr. Yuanyuan (Crystal) Jin is a visionary biotechnology entrepreneur with deep expertise inmolecular pharmacology and structural biology. She is the Co-founder and CEO of BoundTherapeutics, where she has led the development of an innovative RNA therapeutics platformtargeting aggressive cancers and genetic disorders.Dr. Jin earned her Ph.D. from Thomas Jefferson University, where she pioneered a platform toinhibit oncogenic microRNAs. With over a decade of industry experience, she has successfully advanced multiple lead molecules, specializing in tissue-specific RNA drug delivery and AI/MLdrivenpeptide ligand design through the Magic Bullet Designer platform.
In addition to her scientific leadership, Dr. Jin has forged strategic partnerships, negotiated licensing agreements, and secured government funding to support Bound Therapeutics' growth.She is an inventor on multiple patents and has presented her research at leading scientific conferences. Her commitment to innovation continues to drive Bound Therapeutics toward delivering next-generation RNA-based therapeutics.
Dr. Tian is the Vice President of R&D at Sirnaomics Ltd and serves as the General Manager of Sirnaomics (Suzhou). With over 15 years of experience in pharmaceutical research, he currently oversees the corporate development of siRNA novel drugs and related delivery technologies. He earned his Ph.D. in Organic Chemistry from University of Delaware under the guidance of Professor D.F. Taber, and subsequently conducted research in the laboratories of Professor K.C. Nicolaou at The Scripps Research Institute (TSRI) and A*STAR.
I received my Ph.D. degree in 2021 from the Center for Excellence in Molecular Cell Science, Chinese Academy of Sciences (CAS), and continued as a postdoctoral researcher in the same institute. After completing my postdoctoral training in June 2024, I was appointed Assistant Researcher and promoted to Associate Researcher in December 2024. Since my doctoral studies, my research has focused on the functions and applications of small non-coding RNAs, including: (1) development and application of single-cell small RNA and RNA sequencing technologies; (2) functional studies of miRNAs and piRNAs in mammalian oocytes and early embryonic development; and (3) establishment and optimization of the saiRNA drug platform. My work has been published in journals such as Nature Cell Biology, Nature Communications, and Genome Biology. I am also a member of the Youth Innovation Promotion Association of CAS.
Tobias Poehlmann, Biologist by training, did his PhD in cancer biology investigating the effect of silencing genes involved in cell signaling in cancer cells. Involved in the European Network of Excellence “EMBIC” he intensified his knowledge in the field of silencing oligonucleotides and built his scientific network. As an independent researcher he stepped in the field of therapeutic oligonucleotides, developed the technology of peptide-siRNA prodrugs and Co-founded several companies focused on oligonucleotide drug development and GMP manufacturing. Tobias is a university lecturer and enthusiastic oligonucleotides scientist.
Hideaki Sato, MSc. President CEO of Luxna Biotech
Withdrew from Kyoto University Graduate School of Agriculture, Doctoral Program in February 2004, he joined GeneDesign Co., Ltd. (currently Ajinomoto Bio-Pharma Services), a CDMO for nucleic acid medicines, as General Manager, Technical Service and Marketing Division, Executive Officer, and Director (in charge of business), overseeing contract manufacturing business, building a GMP production system, and creating seeds for nucleic acid medicines going for Phase1. He co-founded Luxna Biotech Co., Ltd. in December 2017 and assumed the position of President CEO in February 2018, which he continues to today.
Professor Lin focuses on developing novel strategies to combat RNA viruses, with a particular emphasis on animal influenza viruses and coronaviruses that pose severe threats to human health due to their high mutation and recombination rates, rendering traditional vaccines and drugs insufficient. Centering on the molecular mechanisms of RNA interference (RNAi)-mediated antiviral responses in poultry, Lin elucidated the antiviral molecular mechanisms of the avian RNAi system, and pioneering probiotic-delivered bifunctional antiviral technology.
Lin have presided over 11 research projects, including 2 National Natural Science Foundation projects and 5 provincial/ministerial-level projects, and was selected for the Jiangsu Provincial Excellent Youth Fund. As first or corresponding author, Lin have published 16 SCI papers such as Nucleic Acids Research and Journal of Advanced Research.
Dr. Yamamoto obtained his degree from the Graduate School of Pharmaceutical Sciences at Osaka University in 2012, under the mentorship of Professor Satoshi Obika. After graduating, he became an Assistant Professor of the Obika Lab. In 2015, Dr. Yamamoto broadened his research experience by joining the Martin Schnermann Laboratory at the National Institute of Health in the U.S. In 2018, he was appointed as an Associate Professor at the Graduate School of Biomedical Sciences at Nagasaki University, working in Professor Asako Yamayoshi's Lab. Beyond his academic pursuits, Dr. Yamamoto is also involved in the clinical application development at Liid Pharmaceuticals Inc., an oligo drug startup originating from the National Cerebral and Cardiovascular Center in Japan.
Awards: The Nucleic Acids Therapeutics Society of Japan (NatsJ) Award for Young Scientists (2022)
XU Congcong, Ph.D., serves as an Associate Professor at the International College of Pharmaceutical Innovation of Soochow University. He is a recipient of the Shanghai Pujiang Talent Program and Jiangsu Provincial Young Elite Talent Support Initiative. He earned his B.S. in Chemistry from Hunan University and a Ph.D. in Pharmaceutical Sciences from The Ohio State University College of Pharmacy. During 2020-2023, as the Head of mRNA Platform R&D at a leading mRNA therapeutics company, Dr. Xu co-developed the mRNA AI algorithm LinearDesign (Nature, 2023) in collaboration with Baidu Research USA, and pioneered the circular RNA AI algorithm circDesign ( bioRxiv, 2023). He advanced mRNA-based personalized cancer vaccines into clinical trials in Australia and investigator-initiated trials at Shanghai East Hospital (Science Advances, 2024), and facilitated the overseas clinical development of COVID-19 mRNA vaccines (EClinicalMedicine, 2024). Appointed as a Young Distinguished Professor at Soochow University in 2023, he currently leads or participates in multiple national and municipal research programs, including the National Key R&D Program, Shanghai Science and Technology Innovation Action Plan, and Suzhou Medical Research Projects. He serves as the Young Executive Editor-in-Chief of BME Horizon. Dr. Xu has authored/co-authored numerous high-impact publications in journals such as Nature, Science Advances, Nano Today, and Nano Research as first/corresponding author. His intellectual contributions include 5 granted international patents and 5 pending domestic patent applications related to RNA therapeutics.
Professor Han Xuexiang is a researcher, project leader and doctoral supervisor of the Center of Excellence in Molecular and Cell Science, Chinese Academy of Sciences.
Selected in the national high-level talents (overseas) project and Shanghai Leading Talents (overseas) project.
Hanz Biotechnology (Suzhou) Co., LTD., Co-founder & CSO.
He received his bachelor's degree from Shanghai University in 2014, and his PhD from Tsinghua University in 2019 (jointly trained by National Center for Nanoscience and Technology). From 2020 to 2024, he worked as a postdoctoral researcher in the laboratory of Professor Michael J. Mitchell, University of Pennsylvania, USA, during which he collaborated with Professor Drew Weissman, winner of the Nobel Prize, on the research of LNP-mRNA vaccines and drugs. In February 2024, he joined the Center of Excellence in Molecular and Cell Science, Chinese Academy of Sciences. His research focuses on the construction of novel LNP and RNA drug delivery. He is the first-author (including co-first author) of Nat.Chem., Nat.Nanotech., Nat.Mater., Nat.Commun. He has published nearly 20 papers in other journals, authorized one Chinese patent, and applied for a number of PCT patents.
Dr. Liang Zhang currently serves as a Researcher and Ph.D. advisor at the Hangzhou Institute of Medicine, Chinese Academy of Sciences, and is a founding member of the Vaccine Professional Committee of the Chinese Society of Biotechnology. His research focuses on the design of nucleic acid-based drugs, including mRNA and RNAi. He also specializes in predicting novel tumor antigens and developing advanced RNA structure analysis and prediction algorithms.
Dr. Liang Zhang's work, the LinearDesign algorithm, a groundbreaking mRNA vaccine design tool, was published in Nature in 2023. More recently, he developed circDesign, an innovative algorithm that significantly enhances circular mRNA circularization efficiency, stability, and translatability, as well as in vivo immune responses. He has also made substantial progress in tumor neoantigen discovery and development.
He completed his undergraduate studies at Nankai University and earned two master's degrees, one from the Hong Kong University of Science and Technology and another from London Business School. He received his Ph.D. in Computer Science from Oregon State University in the U.S. Before his current academic role, he worked at Baidu Research in the U.S., focusing on AI-driven drug development and the clinical and commercial translation of drugs. He designed an mRNA COVID-19 vaccine that received emergency use approval in Laos two years ago, and his mRNA cancer and infectious disease vaccines have entered clinical trials or finished animal tests.
Dr. Seigo Kimura is an Assistant Professor at the Integrated Research Consortium on Chemical Sciences, Nagoya University, and a Specially Appointed Assistant Professor at the Institute for Chemical Research, Kyoto University. He received his Ph.D. in Pharmaceutical Sciences from Hokkaido University under the supervision of Prof. Hideyoshi Harashima. Dr. Kimura’s research focuses on the development of lipid-based nanoparticles (LNPs) for the targeted delivery of nucleic acids (DNA, RNA) in vivo, with applications spanning gene therapy, cancer immunotherapy, and vaccination. His work encompasses the design of lipid materials and the compositional optimization of LNPs for enhanced mRNA delivery, as well as the elucidation of the roles of endogenous biological factors in tissue-specific delivery. He has also authored several reviews on LNPs and nucleic acid delivery, contributing to the advancement of this rapidly evolving field. At Nagoya University, within Prof. Hiroshi Abe’s group, his recent work has advanced the functional analysis of emerging nucleic acid modalities, such as chemically modified mRNA and circular RNA, for therapeutic applications in protein replacement, cancer immunotherapy, infectious diseases, and metabolic disorders. This research integrates comprehensive in vitro and in vivo analyses, including tissue- and cell-specific evaluations using animal models. In parallel, at Kyoto University, within Prof. Shiroh Futaki’s group, Dr. Kimura is developing novel delivery systems based on peptides and peptide-lipid hybrid materials, aiming to create innovative polymer and hybrid platforms for the enhanced intracellular delivery of nucleic acids.
Heinrich Haas, Department of Biopharmaceutics and Pharmaceutical Technology Johannes Gutenberg-Universität, Mainz Mainz, Germany. Ph.D. in the group of Prof. Dr. Helmuth Möhwald at Johannes-Gutenberg Universität Mainz. Researched lipid membranes and organized bio-molecular systems. In pharmaceutical industry (Munich Biotech, Medigene, BioNTech) developed different types of nanoparticle products to clinical stage. Focus on advanced approaches for nanoparticle development and control.
Ed Yaworski is the Chief Technology Officer at Genevant Sciences GmbH where he leads the development of nucleic acid therapeutics utilizing Genevant’s proprietary lipid nanoparticle (LNP) technology or next generation ligand conjugate technology. He has more than 30 years of experience in the pharmaceutical industry including over 20 years working with delivery systems for nucleic acids, during which he helped advance more than a dozen products into clinical trials in the areas of oncology, metabolic disorders, and infectious diseases. Ed is an expert in LNP pharmaceutical development, scale-up, lyophilization, and manufacturing. He is a pioneer of LNP formulation methods and compositions and is an inventor of Genevant’s leading LNP delivery technology. Ed holds an MSc in Chemistry from the University of Manitoba.
Dr. Li Tianyao holds a PhD from the National Center for Nanoscience and Technology. His research focuses on the rational design and application of nanostructured lipid nanoparticles (LNPs).
Dr. Yeong-Chan Ahn is the Business Development Director at SML Biopharm, a South Korea–based biotech company specializing in next-generation mRNA-based therapeutics and vaccines. He holds a Ph.D. in Chemistry from the University of Alberta, Canada, and completed postdoctoral research at the University of Texas at Austin. With over 10 years of academic experience in North America, Dr. Ahn brings global expertise in mRNA therapeutics, technology partnerships, and strategic investment. At SML Biopharm, he leads strategic collaborations and international joint R&D initiatives, including mRNA-based antibody therapies and immuno-oncology pipelines. Dr. Ahn plays a central role in expanding the company’s global network and advancing commercialization strategies through BD-driven innovation.
Dr. Ni Qianqian is a tenure-track Assistant Professor at National University of Singapore. She obtained her MD and PhD from Nanjing University, China. She did PhD and postdoc research training at National Institutes of Health (NIH), US before joining National University of Singapore. Dr. Ni’s research has been dedicated to advancing the application of nanotechnologies in RNA delivery. Her work focuses on two key areas: 1) the development of nucleic acid nanomedicines for cancer immunomodulation and theranostics, and 2) the innovation of lipid nanoparticle technologies and RNA modifications to enhance protein production.
Dr. Jiang Xu serves as Vice R&D Director at Zcapsule Inc. (Shanghai). He is a scientist and innovator with a focus on microfluidics, mRNA-LNP nanomedicine, and advanced drug delivery systems. Holding a cotutelle Ph.D. in Chemical Engineering (University of Waterloo) and Physical Chemistry (University of Bordeaux/CNRS), he completed postdoctoral training at Harvard Medical School, Toronto Metropolitan University, and PSL-ESPCI ParisTech. He has won a series of competitive fellowships and grants, including NIH RADx Grant (2020–2021, 1% funding rate), National Overseas High-Level Youth Talent Program (2022-2025), and Shanghai Rising-Star Program (2023-2026).
Dr. Xu has led transformative projects in biopharmaceutical R&D, such as China’s first Omicron-specific mRNA vaccine (Phase II clinical trials, 2023), the world’s first mRNA-based monkeypox vaccine (published in Nature Communications, 2023), and establishment of a GMP-compliant LNP production platform (2 billion doses/year). His interdisciplinary expertise bridges academia and industry, combining scientific rigor with a vision for scalable, patient-centric innovations in global healthcare.
Responsible for providing technical support for mRNA projects and serving mRNA clients across China. With extensive experience in product development and product management. Possesses in-depth expertise in addressing process flow challenges, translating product functionalities into solutions, and working with downstream hardware products. Previously worked at Bayer France and gained substantial product and process experience at Cytiva China.
Dennis Yu is the founder and CEO of Value Biologics. Dennis received PhD and MBA degrees. He has over 30 years of global biopharma experience in R&D and biomanufacturing. Prior to founding Value Biologics, he has been working for big pharma and biotech including Novartis, BMS and Wuxi Biologics. He has led R&D teams to develop and manufacture over 30 biopharmaceuticals covering vaccines, antibodies, protein drugs and nucleic acid drugs, some of which have been regulatorily approved and commercialized successfully. In Value Biologics, he’s been working on advanced mRNA-LNP platforms including circular mRNA and organ targeting LNP to develop novel vaccines, therapeutics, cell and gene therapies for unmet medical needs.
Graduated with a Ph.D. in Bioinformatics from the University of Science and Technology of China in 2014, under the guidance of Professor Liu Haiyan. Many years of experiences in molecular dynamics simulation and high-throughput sequencing, and have served as the Director of Bioinformatics for Micro Spin Genes and the Head of Nucleic Acid Drug Development for Yidu Cloud. Successfully developed multiple clinical high-throughput data analysis platforms and have authored multiple invention patent authorizations and SCI papers. As the co-founder of Therarna, responsible for identifying and optimizing upstream targets of mRNA drugs, leading the team to develop a systematic platform for identifying cross species cellular immune epitopes, designing humoral immune antigens, and optimizing mRNA sequences.
Jin Li is Head of the Research Department at CanSino (Shanghai) Biological Research Co., Ltd., where she leads research and development in mRNA vaccine and therapeutic innovation. She graduated from Columbia University with a degree in Biomedical Engineering, and has over nine years of experience in the biopharmaceutical industry.
Jin has been with CanSino for eight years, and was among the first scientists in China to enter the mRNA vaccine field, playing a foundational role in building the company’s mRNA R&D capabilities from the ground up.
She was the chief designer of CanSino’s monovalent and bivalent COVID-19 mRNA vaccines, CS-2034 and CS-2602. Both candidates received IND approvals and successfully completed five clinical trials, demonstrating strong safety profiles, robust immunogenicity, and protective efficacy.
Currently, her focus includes the development of inhalable mRNA vaccines, as well as expansion into prophylactic and therapeutic human vaccines, veterinary vaccines, and mRNA-based therapeutic products. She is also spearheading innovation in self-amplifying RNA platforms and the ISL-3C-LNP delivery system, with the goal of improving expression efficiency, safety, and immune response.
Linping Wu is a Principal Investigator and Ph.D. students Supervisor at the Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences. Prior to returning to China, he earned his Ph.D. from the University of Copenhagen, Denmark, and completed postdoctoral training there. His research focuses on nucleic-acid therapeutics and vaccines, as well as on nanotechnology-enabled targeted delivery systems. Dr. Wu serves as the Chief Scientist for the Strategic Priority Research Program of the Chinese Academy of Sciences in the field of mRNA drug, leads projects under the National Key R&D Program, and heads sub-tasks of the National Major New Drug Innovation Initiative. He has published more than 80 papers in peer-reviewed international journals, with a cumulative impact factor exceeding 600 and over 5,000 citations. He has been invited to contribute 10 book chapters and has filed or been granted more than 20 patents.
Dr. Getts is the CEO and co-founder of Myeloid Therapeutics, a clinical-stage immunology company based in Cambridge. Under his leadership, Myeloid Therapeutics has raised over $200 million in combined equity and non-dilutive financing to support the translation of cutting-edge mRNA immune cell programming therapies to the clinic. Myeloid is renowned as the first company in the world to deliver in vivo CAR therapy to humans. Dr. Getts is also the founder and interim CEO of Aurora Biosynthetics, a Myeloid subsidiary focused on GMP DNA & RNA manufacturing, based in Sydney, Australia.
Prior to founding Myeloid, Dr. Getts was the Vice President of Research at TCR2 Therapeutics (NASDAQ: TCRR), where he was a pivotal member of the leadership team that successfully secured a Series B financing round of $120 million and an IPO of approximately $80 million.
Before his tenure at TCR2, Dr. Getts was the founder, inventor, and Chief Scientific Officer of Cour Pharmaceuticals Development Company. Cour is a nanotechnology platform company dedicated to addressing autoimmunity and inflammation. During his time there, Dr. Getts was instrumental in company formation, development and execution of clinical programs and negotiating several pharmaceutical collaborations and licenses, including a significant partnership with Takeda, which licensed TIMP-GLIA (TAK-101) for $420 million plus royalties.
Earlier in his career, Dr. Getts served as the Director of Research & Development at Tolera Therapeutics. In this role, he was the lead immunologist responsible for advancing the company’s monoclonal T-cell antibody program from discovery to Phase 3 clinical trials.
Dr. Getts has received numerous honors and awards and holds over 100 patents and patent applications. Since 2010, he has played a direct or indirect role in bringing nine novel RNA, cell, and immune products from discovery to the clinic. He is widely published, with over 50 peer-reviewed publications in prestigious journals, including Nature Biotechnology, Science Translational Medicine, and Nature Communications.
Dr. Getts completed his postdoctoral training at Northwestern University in Stephen D. Miller’s laboratory. He holds a PhD in Medicine from the University of Sydney and an MBA from Western Michigan University. His extensive expertise and groundbreaking contributions to the fields of immunology and RNA therapeutics make him a distinguished leader and innovator in the biotech industry.
Dr. Yang Wenbing, Ph.D. from Sun Yat-sen University, with over 10 years of research experience in RNA editing technology. In 2020, he published an original article on the irCLASH technology in 《Nature Structural & Molecular Biology》, which was the first global study to reveal the substrate structure and editing mechanism of the human endogenous RNA editing enzyme ADAR, with a focus on its substrate preference and high editing efficiency. In 2025, he published a paper in 《Nature Biotechnology》 on an RNA editing system based on the structure of endogenous ADAR substrates, providing a theoretical foundation for the application of RNA editing technology in clinical drug development. Dr. Yang is currently the R&D Director at RecoRNA, where he is responsible for building the company’s RNA editing platform, drug design and screening, pharmacology and efficacy research, and patent strategy. He has obtained three national invention patents and has submitted over ten invention patents.
Zhu Xinye (Judy Zhu), Resident Physician, Liver Surgery Department, Renji Hospital, Shanghai Jiao Tong University School of Medicine. Research focuses on innovative therapies for genetic metabolic diseases, with emphasis on mRNA/LNP platforms and circRNA applications. Representative work includes Preclinical Evaluation of AGT mRNA Replacement Therapy for Primary Hyperoxaluria Type I Disease.
Dr. Yuhong Cao is a scientist and principal investigator at the National Center of Nanoscience and Technology, Beijing, China. She earned her B.S. in Chemistry from Linfield College in Oregon, USA, and her Ph.D. in Materials Science and Engineering from Stanford University, California. Under the mentorship of Professor Nicholas Melosh, her doctoral research focused on developing a nondestructive nanostraw system for longitudinal living cell sampling. Following her Ph.D., Dr. Cao pursued postdoctoral training in T cell engineering by applying CRISPR-Cas technology. She worked with Professor Peidong Yang, in partnership with Professor Jennifer Doudna at the University of California, Berkeley. She then returned to Stanford University for a second postdoctoral fellowship with Professor Steven Chu, focusing on in vivo single-molecule tracking. At the National Center of Nanoscience and Technology, Dr. Cao leads a research group specializing in Nanomaterials and Plant Cell Surface Interfaces. Her work aims to develop universal intracellular delivery systems for natural plants to accelerate plant breeding. By exploring nanomaterials to facilitate effective gene editing using CRISPR technology, her lab addresses the critical need for efficient delivery systems in plants. This research has the potential to significantly shorten plant breeding cycles, enabling ultra-fast breeding and advancing agricultural innovation.
Dr. Liqiang FENG received his Bachelor of science from Nanjing University in 2005. He received a Ph.D. in Biotechnology and Biomedicine from Guangzhou Institutes of Biomedicine and Health, CAS, in 2010. He served as a Research Assistant there from 2010 to 2012, a Research Associate from 2013 to 2018. From 2019 to present, he served as a Principal Investigator in Guangzhou Institutes of Biomedicine and Health, CAS. Dr. Feng has over 40 scientific publications in infectious diseases and vaccine. His recent research interests include circular RNA technology and recombinant viral vectors for vaccines against mosquito-borne flaviviruses. His group have reccieved several grants from NSFC and CAS.
Xueliang Yu is the Vice President of Starna Therapeutics, responsible for the company's delivery and chemical departments. He is mainly engaged in the research of nucleic acid delivery technology of non-liver-targeted liposome (LNP)/polymer nanomaterials. He graduated from Tianjin University with a bachelor's degree and a Ph.D. in organic chemistry from UTSW, and worked as a postdoctoral researcher at UTSW Cancer Center, publishing more than 20 papers/patents. He has many years of experience in organic total synthesis, lipid synthesis and LNP target design.
Dr. Xian Zeng, the Co-Founder and CEO of Byterna Therapeutics, leads this enterprising startup at the vanguard of groundbreaking circular mRNA-based immunotherapies. Holding a PhD in pharmaceutical science from the National University of Singapore, he previously served as Associate Professor at Fudan University School of Pharmacy, spearheading interdisciplinary research in AI-empowered mRNA drug discovery. Under his visionary leadership, Byterna is pioneering SEAA (Safe, Effective, Accessible, Affordable) CAR-T therapies for global patients, leveraging proprietary circular mRNA platforms and targeted delivery systems.
Zefeng Wang is a chair professor of Southern University of Science and Technology, and the scientific founder of Shanghai CirCode Biomedicine Inc. His research focuses on the regulation of gene expression in RNA level. His group uses bio computational and experimental approaches to study RNA regulation in a systematic fashion, and developed artificial proteins to specifically manipulate RNA metabolism. He has discovered the noncanonical translation of circular RNAs (circRNAs) in human cells, which promoted the use of circRNA as new generation of mRNA therapy. His work was recognized by many research awards, including RNA Society/Scaringe Young Scientist Award, Alfred Sloan Research Fellow, Beckman Young Investigator, Max-Planck Fellow, CAS pioneer hundred talents program (type A), etc. Based on the research from his lab, he co-founded the CirCode BioMed Inc that seeks to develop circRNA drugs for various human diseases.
Ph.D. Old Dominion University, Virginia. USA, concentrated on vector-borne disease.
ORISE fellow, The US Food and Drug Administration, CBER,DVP, OVRR.
An expert on Flaviviridae vector-borne diseases including LGTV, ZIKV, DENV infection and transmission through the facilitation of exosome and published on several top journals (PNAS, EMI, PLoS Pathog, etc.).
ORISE fellow at the FDA, worked on live attenuated Rubella vector vaccines expressing RSV, Covid-19 or HIV interest proteins.
Now working as a leading scientist & academic secretary of CNBG-Virogin, leading POC research team and be responsible for all the infectious disease mRNA vaccine pipelines that under the POC stage.
Dr. Pankaj Trivedi obtained his doctorate from Karolinska Institute, Sweden. Currently, he is a Professor of Laboratory Medicine at the faculty of Medicine, Sapienza University in Rome, Italy. Dr. Trivedi has also served as visiting professor, BIDMC, Harvard Medical School, Boston. Over the last 30 years, his main interest has been to study the role of Epstein-Barr virus (EBV) in pathogenesis of B cell lymphomas. The Trivedi lab has made some pioneering contributions how EBV through its latent proteins alters cellular microRNAs for immune evasion. He is a coinventor of the RNA aided Immunotherapeutics related patent and has authored over 70 papers in this field.
Eleni Anastasiadou received her Ph.D. from La Sapienza, University of Rome, Italy, specializing in microbiology and virology. She focused on the interactions of cellular miRNAs with Epstein Barr Virus latent proteins in lymphoma. Her pioneering work led to the identification of how viral proteins subvert cellular miRNAs and contribute to cancer. During her work at Beth Israel Deaconess Medical Center/Harvard Medical School, Boston has developed an RNA-aided cancer immunotherapy method and a bench-to-bedside delivery of miRNAs in lymphomas. Currently, she is an Assistant Professor at La Sapienza University, focused on the role of miRNAs in regulating immune responses in cancer.