In Vivo CRISPR Medicine: From Scientific Promise to Patients at Scale

In vivo genome editing is entering an important new phase. Advances in editing technologies, delivery platforms and translational science are bringing the prospect of one-time, directly administered genetic medicines closer to patients, while a growing pipeline of programmes is testing what these approaches can achieve in the clinic. At the same time, significant challenges remain, from achieving efficient and tissue-specific delivery to demonstrating safety, durability and scalability across different diseases and patient populations.

Co-organised by CRISPR Medicine News and the Chinese Society of Gene & Cell Therapy (CSGCT), this special symposium will bring together leading scientists and industry experts from China and Europe to explore the current state and future direction of in vivo genome editing. Through presentations spanning therapeutic development, liver-directed approaches and next-generation RNA delivery, followed by a joint panel discussion, the symposium will examine recent progress, the scientific and translational challenges that still need to be addressed, and how greater exchange between the Chinese and European gene-editing communities can help move the field from scientific promise towards patients at scale.

In this symposium, you will learn about:

• Perspectives on the development of in vivo genome editing in China, including emerging technologies, translational progress and opportunities to advance new genome-editing medicines towards the clinic. (Yongzhong Wang, AccurEdit Therapeutics)
• Next-generation RNA delivery for advanced CRISPR modalities, including technologies designed to support efficient, clinically applicable in vivo delivery and enable increasingly sophisticated genome-editing approaches. (Christine Duthoit, RNAlead)
• The development of one-and-done genetic medicines for inherited metabolic diseases, including liver-directed approaches and the challenges involved in translating these therapies from research towards patients. (Alessio Cantore, San Raffaele University & San Raffaele–Telethon Institute for Gene Therapy)
• Advances in in vivo genome-editing technologies and their translation into new therapeutic approaches, with insights into the scientific and technical developments helping move the field forward. (Rui Tian, GeneRulor Co., Ltd.)

Webinar Programme (CEST / China Standard Time)

13:00 / 19:00 Welcome and introduction by Jens-Ole Bock, Founder, CMN and Karen O´Hanlon Cohrt, Intelligence Manager, CMN

13:05 / 19:05 Yongzhong Wang, AccurEdit Therapeutics
Key Considerations in Developing LNP-Based In Vivo Gene Editing Therapies for Rare Diseases and Under-Treated Chronic Conditions

13:25 / 19:25 Christine Duthoit, RNAlead
A Clinic-Ready RNA Delivery Platform Supporting Advanced CRISPR Modalities (FlashRNA®)

13:45 / 19:45 Alessio Cantore, San Raffaele University & San Raffaele–Telethon Institute for Gene Therapy
Towards One-and-Done Gene Therapies for Defects of Hepatic Metabolism in Pediatric Patients

14:05 / 20:05 Rui Tian, GeneRulor Co., Ltd.
Off-Target Detection and Regulatory Submission Strategies for Gene Editing

14:25 / 20:25 Panel discussion with all four speakers
Discussion on the current state of in vivo genome editing, remaining scientific and translational challenges, and opportunities for greater exchange and collaboration between China and Europe.

14:55 / 20:55 Closing remarks by CRISPR Medicine News and CSGCT

15:00 / 21:00 Close

Yongzhong Wang, PhD, Founder, Chairman and CEO, AccurEdit Therapeutics, China

Key Considerations in Developing LNP-Based In Vivo Gene Editing Therapies for Rare Diseases and Under-Treated Chronic Conditions

Dr. Yongzhong Wang, Founder, Chairman and CEO of AccurEdit Therapeutics, has more than 20 years of proven experience in the R&D, industrialization, commercialization, and internationalization of innovative biopharmaceuticals in China and overseas. He also has extensive experience in successfully managing and operating pharmaceutical companies on both the sponsor and service-provider sides.

Dr. Wang previously served as CEO of CMAB Biopharma, President of Pharmaceuticals at Simcere Pharmaceutical Group, CEO of CONHON, and Scientist at Genzyme Corporation in the United States. Dr. Wang holds a Ph.D. from Tufts University, and his publications have been cited more than 4,000 times to date.

Meet AccurEdit Therapeutics and one of their scientists, Songyuan Li, at CRISPRMED27, April 19-22, 2027 in Copenhagen, Denmark.

Christine Duthoit, Co founder – CEO – CSO, RNAlead, France

A Clinic-Ready RNA Delivery Platform Supporting Advanced CRISPR Modalities (FlashRNA®)

PhD scientist with extensive research experience in France (INSERM, CNRS) and the U.S. (St Jude Children’s Research Hospital), Christine has spent 17 years in the biotech industry. She has a strong interest in immunotherapy breakthroughs and spent 16 years at Flash Therapeutics managing gene transfer projects, from cell to in vivo models and finally clinic, as CSO.

As the CEO/CSO at RNAlead, she is dedicated to advancing transformative cell and gene therapies thanks to the FlashRNA® technology.

Alessio Cantore, Associate Professor of Tissue Biology at San Raffaele University and Group Leader at the San Raffaele – Telethon Institute for Gene Therapy in Milan, Italy

Towards one-and-done gene therapies for defects of hepatic metabolism in pediatric patients

Alessio Cantore is an Associate Professor of Tissue Biology at San Raffaele University and a Group Leader at the San Raffaele – Telethon Institute for Gene Therapy in Milan, Italy. He oversees the liver gene therapy research unit and supervises a team of more than 10 people. His research interests include lentiviral vector engineering, liver tissue dynamics, and the translational applications of liver-directed gene transfer and gene editing. Recently, his work has focused on two related but distinct areas: one more fundamental, investigating liver stem cells and tissue dynamics, and another more translational, concerning inherited hepatic metabolic diseases.

He began his laboratory training at the University of Bologna and continued as an undergraduate at the San Raffaele Telethon Institute for Gene Therapy. He was then admitted to the international Ph.D. Program in Cellular and Molecular Biology. During his Ph.D. and postdoctoral training, he focused on liver-directed lentiviral gene therapy, leading projects from preclinical studies to non-human primates, vector engineering, and efficacy/safety assessments. He is a scientific co-founder of GeneSpire, a start-up aiming to pre-clinically and clinically develop liver-directed lentiviral gene therapy for the treatment of rare inherited diseases affecting liver function, backed by Sofinnova investment funds.

Dr. Rui Tian, Chief Technical Officer, Generulor Co., Ltd., China

Off-Target Detection and Regulatory Submission Strategies for Gene Editing

Rui Tian received her Ph.D. from Sun Yat-sen University and has long pursued a coherent line of research on gene editing and gene therapy. As principal investigator she has led one Young Scientists Fund project of the National Natural Science Foundation of China, one project of the Postdoctoral Innovative Talent Support Program, two General Program grants of the China Postdoctoral Science Foundation, one Youth Fund project of the Guangdong Provincial Natural Science Foundation, and one Distinctive Innovation Project for Young University Teachers. Committed to the clinical translation of gene editing, she has served as a core member of one clinical trial and has filed 20 Chinese national patent applications, 15 of which have been granted. As first and/or corresponding author she has published 15 SCI papers in leading international journals, including BMJ, Science Advances, Nucleic Acids Research, Med, Nature Communications (2 papers), Cell Genomics, Briefings in Bioinformatics, Small and Clinical and Translational Medicine; nine of these papers appeared in journals with an impact factor above 10. She was nominated for the Future Female Scientists Program in 2020 and has been appointed adjunct master’s supervisor at Tongji Medical College, Huazhong University of Science and Technology. In recent years her work has centred on translating research findings into clinical practice, including the development of new high-throughput technologies for in vitro and in vivo off-target detection and the creation of novel CRISPR systems with independent intellectual property rights. Drawing on extensive experience in the safety assessment of gene editing, she has supported several cell and gene therapy companies in completing IND submissions to the FDA and the CDE.

CRISPR Medicine News

CRISPR Medicine News (CMN) is the world's leading platform for CRISPR and genomic medicine, with a monthly readership of 35,000 across 170 countries. Since 2018, CMN has tracked CRISPR's journey from groundbreaking research tool to clinical reality, delivering weekly news, expert interviews, webinars and trial updates to its global audience. CMN also convenes the field in person through CRISPRMED, an annual conference launched in 2024 that unites the global CRISPR medicine community to explore the latest advances, ongoing challenges, and emerging solutions in the field. In 2026, CMN expanded its offering with the launch of CMN Intelligence, an extensive open-access database combined with quarterly reports tracking the global landscape of gene-editing trials. At its core, CMN is committed to speeding up the clinical translation of safe gene-editing therapies, helping bring new treatment options to patients worldwide facing rare diseases and complex cancers.

CSGCT

The Chinese Society of Gene & Cell Therapy (CSGCT) is a leading professional non-profit organisation dedicated to advancing innovation and collaboration in the gene and cell (GCT) field. By connecting scientists, clinicians, regulators, industry leaders, and investors, CSGCT accelerates the translation of breakthrough research into real-world therapies. The society also promotes global integration and expands access to innovative treatments, with the ultimate goal of improving patient outcomes.

CRISPR Medicine News and the Chinese Society of Gene & Cell Therapy Announce Media Partnership - Press Release August 3rd, 2026

Meet CMN on WeChat