On September 06, 2024, GenAssist Ltd (GenAssist), announced that the first DMD patient has been successfully dosed with its base editing drug, GEN6050X injection, in an investigator-initiated trial (IIT). “To date, no gene editing drug has entered clinical trials for DMD indication. This first-in-human dosing marks that DMD treatment has entered the gene editing era”, […]
On July 29, 2024, Suzhou GenAssist Therapeutical Co., Ltd, the operation entity of GenAssist Ltd (GenAssist), announced that Pre-IND (PIND) meeting request for its first base editing DMD drug, GEN6050X, has be submitted to NMPA. Previously, the PIND meeting to FDA was held in April 2023. The PIND filing to FDA and NMPA indicated the
On May 30, 2024, Suzhou GenAssist Therapeutical Co., Ltd, the operation entity of GenAssist Ltd (GenAssist), announced that the IIT(NCT06392724) kickoff meeting for its first base editing DMD drug, GEN6050X, has be successfully held in Peking Union Medical College Hospital (PUMCH). This program is sponsored by Suzhou GenAssist Therapeutics and led by Professor Yi Dai,
GenAssist Therapeutics successfully completed a virtual meeting with the TREAT-NMD Advisory Committee for its first base editing product on September 29, 2023. Eighteen representatives, including distinguished preclinical and clinical experts in Duchenne Muscular Dystrophy (DMD) therapy, as well as DMD representatives attended this meeting. A comprehensive review and advice were received on November 13 after
Recently, the team led by Professor Xing Chang, West Lake University, published an article titled “Efficient exon skipping by base-editor-mediated abrogation of exonic splicing enhancers” in Cell Reports. In this paper, Han Q. et al employed Targeted AID-mediated mutagenesis (TAM) cytosine base editor (CBE-TAM) to edit various hotspot mutations of DMD gene in vitro. High
On July 9, 2021, GenAssist has successfully closed a multi-million RMB Pre-Series A financing , bringing the total raised funds to nearly 100 million RMB. The round was led by Sequoia Capital China Fund, with participation from Westlake University Industrial Investment, Suzhou New District Industrial Investment, and the Wang Jiaquan Family Fund. Existing shareholder of
Recently, GenAssist, which is the first company in China to develop gene therapy drugs using base editing technology, announced the closing of a multi-million dollar Series A financing. The round was led by AstraZeneca-CICC Healthcare Industry Fund, with participation from Baidu Ventures (BV), a well-known equity investment institution, and existing shareholder Sequoia China, who continued
GenAssist Completes Multi-Million Dollar Series A Financing Read More »
Recently, Suzhou GenAssisit Therapeutics Co.,Ltd. (hereinafter referred to as “GenAssist”) announced that its first base editing product, GEN6050, has submitted a pre-investigational new drug (Pre-IND) application to the FDA and has been accepted. GEN6050 is an in vivo base editing drug targeting exon 50 skipping for the treatment of Duchenne Muscular Dystrophy (DMD). The drug
Pre-IND application of GenAssist’s First DMD Drug was Accepted by the US FDA! Read More »
近日，新芽基因创始人兼CEO何春艳博士接受了药明康德内容团队关于碱基编辑技术应用的专项访谈。 作为一项新兴技术，碱基编辑技术有望为疾病治疗带来哪些重大突破？该技术当前面临哪些亟待解决的瓶颈和挑战？新芽基因的技术又有哪些独特之处？对此，何春艳博士给出如下看法。 药明康德内容团队：在新芽基因目前所致力的杜氏肌营养不良疾病领域，当前治疗方式或临床解决方案还面临哪些关键挑战？ 何春艳博士：新芽基因首个产品的适应症为杜氏肌营养不良（DMD基因变疾病。DMD是以肌肉退化和衰弱为特征的神经肌肉进行性疾病，它的发病原因是DMD基因突变造成抗肌萎缩蛋白（dystrophin）表达缺失，引发骨骼肌和心肌的严重缺陷。目前，DMD没有治愈的方法，皮质类固醇药物通常被用来延缓DMD疾病进程，但这些激素类药物的治疗效果并不显著，且副作用较大。随着基因治疗技术的发展，越来越多针对DMD的治疗手段逐步出现。当前，针对DMD的主流基因疗法是反义寡核苷酸（ASO）药物和腺相关病毒（AAV）基因替代疗法。ASO药物是通过调节细胞内的mRNA剪接作用，实现DMD基因外显子的跳跃，产生截短而有功能的抗肌萎缩蛋白来治疗疾病。这类疗法的主要问题是需要反复给药，且在生物体中的利用度较低，不能高效且持续地提高患者的抗肌萎缩蛋白表达量。AAV基因替代疗法可以递送截短的抗肌萎缩蛋白基因的表达框，在肌纤维中过表达这种微型抗肌萎缩蛋白，达到治疗的目的。然而，AAV的基因组不会整合宿主基因组，而是以染色体外基因的形式存在，很可能随着时间的延长被代谢，或随着细胞的分裂被稀释，因此维持长期表达不容乐观。总体而言，DMD疾病突变的抗肌萎缩蛋白大、基因突变位点多，基因治疗药物开发的难度非常大。另外，DMD是一种进展性疾病，疾病的后续进展伴随着肌肉组织的纤维化、炎症等，目前也还没有很好的手段可以逆转这一趋势。
专访丨新芽基因CEO何春艳：手握基因组“铅笔和橡皮”，我们能为患者带来什么？ Read More »
Senior pharmacology and toxicology advisor
Pharmacology, Shang University of Traditional Chinese Medicine.
Senior Vice General Manager at Shanghai InnoStar Biotechnology Co., Ltd.
More than 20 years in preclinical pharmacology and toxicology evaluation of new drugs.
Senior Scientific Advisor
Ph.D., Molecular Cell Biology and Bioengineering, Max Planck Research School.
Senior Director of genome engineering department, AstraZeneca, Gothenburg, Sweden.
More than 10 years’ experience in gene editing tool and drug development.
Senior Clinical Physician Advisor
Ph.D. , Peking Union Medical College, Tsinghua University.
Associate Chief physician. Neurology Department. Peking Union Medical College Hospital, Beijing.
Member of the Neurology Branch of the Chinese Medical Association.
Member of the DMD/BMD Study Group of the Chinese Rare Disease Alliance.
Specializes in complex and rare neurological disorders, gene diagnosis of monogenic.
Senior strategic Advisor
Over 30 years of experience in drug discovery, clinical translation, clinical practice, and operational management Former President of Chongqing Medical and Pharmaceutical College.
Ex-Vice President of the Technical Center at Fosun Pharma.
Senior Biostatistics Advisor
Ph.D. Biostatistics, University of Michigan, USA.
Bettyann Asche Murray Distinguished Professor, Deputy Chair.
Department of Biostatistics, University of Texas, MD Anderson Cancer Center.
Inventor of Bayesian Optimal lnterval PhaseⅠ/ⅡTrial Design (BOlN12); Participated in clinical trial design of many anti-cancer treatment.
Co-funder, Senior scientific Advisor
Ph.D. , Pathology, Ohio State University
Associate Professor at West Lake University, Young Scientist in the 973 Program
Thousand Youth Talents Program, National Outstanding Young Scientist
Inventor of Targeted AID-mediated Mutagenesis (TAM) base editor
CEO, Co-Founder
Ph.D. , Immunology, Shanghai Jiao Tong University.
MBA, Manchester Business School.
Over 20 years of experience in pharmaceutical industry, covering R&D, BD, Investment and Incubation.
Quality management advisor
Former Director of the Quality Assurance Department and Laboratory at the Shanghai Institute of Biological Products
Research and establishment of quality standards for various viral bioproducts as well as registration applications
Committee Member of the Chinese Pharmacopoeia Commission,
Expert Member of the Shanghai Science and Technology Commission
Committee Member of the Quality Management Committee of the Shanghai Medical Industry Association
Member of the New Technology/Skills Professional Committee.