Witnessing Multiple 'Firsts': Highlights from the 6th Gene Editing and Gene Therapy Technology Symposium

Nie Liya, Co-founder, Director, and Executive Vice President of NorthlandFocusing on China’s first gene therapy drug for ischemic ulcers, he provided a detailed explanation of the disease mechanism and the current status of drug development, pointing out that naked plasmids have become the preferred modality for angiogenic therapy due to their unique advantages. He also shared the development history, structural characteristics, clinical research overview, and core data of Sedomingji Injection, fully demonstrating its outstanding efficacy and safety. The success of Sedomingji Injection has filled the gap in the treatment of critical limb ischemia（CLI）...gaps in the fields of sexual diseases and angiogenic drugs, effectively meeting the urgent needs of clinical patients.

Lonza APAC SME Fang QiangyiHighlighting the core philosophy of “Winning at the Starting Line,” it emphasizes that high-quality starting materials are the key guarantee for CGT therapies throughout the entire process from early-stage R&D to GMP-compliant commercialization, with a particular focus on Lonza TheraPEAK.^®TheraPEAK in the Product Matrix^® 293-GT^®Culture Medium, TheraPEAK^®T-VIVO® Culture Medium and Flagship Product TheraPEAK^® AmpliCell^®Cytokines. He pointed out that culture media products feature defined compositions and high batch-to-batch consistency, making them suitable for various process scenarios, while cytokines offer biological activity comparable to that of natural human cytokines along with the advantage of stable inter-batch reproducibility. Following the live broadcast, he addressed online questions from viewers, further demonstrating his professionalism.

Lingyi Biotech CEO Lin QingUnder the theme “Research and Development Progress of Next-Generation Gene Therapy Technology AAV 2.0,” he shared the current industry landscape and technological breakthroughs in gene therapy for monogenic hereditary diseases. He noted that one of the core challenges in rare disease research is the lack of animal models that can adequately simulate disease phenotypes. Meanwhile, he summarized industry trends from the ASGCT Annual Meeting, such as the continuously increasing proportion of gene therapies and the emergence of the central nervous system (CNS) field as a hotspot for R&D. He further provided an in-depth analysis of the R&D distribution of engineered AAV serotypes, the biodistribution characteristics of AAV vectors, and key clinical risk considerations. Additionally, he presented progress on representative pipeline candidates, highlighting LY-M001, the first candidate AAV gene therapy in China targeting Type I Gaucher disease, which has demonstrated favorable efficacy and safety profiles in clinical studies.

Chen Lijing, COO and Vice President of JinweikeShe pointed out that the global gene therapy market is experiencing rapid growth, with indications expanding from rare diseases to common conditions. Ophthalmology has emerged as a hot sector, where the core optimization focus lies in capsid selection and gene sequence design. Regarding pipeline progress, she highlighted two core products: JWK002 and JWK001. The former has been optimized in terms of AAV serotype and viral gene expression cassette; it is the first gene therapy candidate for X-linked retinoschisis (XLRS) in China and the only gene therapy worldwide to have demonstrated therapeutic effects on both retinal structure and function in clinical settings. The latter, targeting neovascular age-related macular degeneration (nAMD), has entered Phase II clinical trials with significant efficacy observed. Addressing industry commercialization pain points, she analyzed the current landscape of high pricing for marketed products and proposed strategies to break through these barriers. Jinweike’s globally first two-plasmid production process can substantially reduce costs, facilitating future commercialization.

Wang Gang, Founder and CEO of Buyang GeneUnder the theme “From PRG Motif to H-Switch: Directed Evolution and NHP Validation of MyoCAP Muscle-Targeting Capsids,” he shared advances in the AAV capsid directed evolution technology platform and its applications. He introduced the comprehensive end-to-end platform for AAV capsid directed evolution—DynEvolib (Dynamic Evolution Library)—and elaborated on its core modules, including PEARS, CALIBRATOR, CAMEL, and CAPTURE, along with practical case studies.