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

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. Presentation title to be announced
• 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

Register here.