Gene Therapy Workshop Event Recap
Gene Therapy Workshop, co-organized by CABS (the Chinese American Biopharmaceutical Society) Science & Technology Committee and CCBEA (California Chinese Biotech/Biopharmaceutical Entrepreneur Association), was successfully held on October 5, 2019 at Hanhai BioLabs in Burlingame, CA. Nearly 150 professionals attended the workshop that included 5 talks given by gene therapy experts from both industry and academia. The topics covered lentivirus gene therapy, Adeno-Associated Virus (AAV) gene therapy manufacturing, and non-viral gene deliver using nanoparticles.
Dr. Yan Wang, the Past President of CABS gave welcome remarks and introduced CABS briefly. Dr. Shenjiang Liu, the President of CCBEA, introduced the history of CCBEA and reviewed the main activities of CCBEA in the past years. Dr. Xiang Yi and Dr. Ken Zhang co-chaired this workshop.
Dr. Gary Li, the founder and CEO of ALSTEM, presented the history of viral gene therapy, from concept formation and early clinical attempts to recent successful clinical applications. He discussed the use of lentivirus as a gene delivery tool and provided details of different generations of the lentivirus packaging systems. Lentiviral approach was recognized as a valuable tool in both ex-vivo and in-vivo clinical applications such as, β-thalassaemia, cerebral adrenoleukodystrophy, and chimeric antigen receptor-T cells.
Dr. Haifeng Chen, CEO of Virovek, Inc., focused on the development of AAV in large scale of manufacturing and process for clinical application. Two major challenges for AAV manufacturing are its productivity and infectivity. Dr. Chen tackled these problems by introducing an artificial intron in the AAV vector and developed a serum free BAC-TO-AAV system with high infectivity and productivity. Under his leadership, Virovek now has the capacity to provide purified AAV vectors exceeding 1 x 10^17 vg scale per production run.
Dr. Niren Murthy, Professor in the Department of Bioengineering at UC Berkeley, shared non-viral technologies and biomaterials developed by his lab for gene delivery. He assembled a delivery vehicle termed CRISPR-Gold that composed of gold nanoparticles complexed with the Cas9/gRNA ribonucleoprotein (RNP), donor DNA, and an endosomal disruptive polymer. The CRISPR-Gold enabled the correction of DNA mutation that causes Duchenne Muscular Dystrophy (DMD) in mdx mice via homologous DNA recombination.
Dr. Kee-Hong Kim, Senior Vice President, Manufacturing & Technical Operations of Tenaya Therapeutics, talked about manufacturing clinical AAVs at scale for gene therapy. He discussed strategic questions for AAV manufacturing, considerations for clinical AAV manufacturing and regulator’s expectation for gene therapy CMC, as well as suggestions for developing a start-up gene therapy company. His suggestions included talent acquisition in AAV manufacturing, compliance of gene therapy CMC, and strategic positioning of products in the market.
Finally, Dr. Shengjiang Liu, Chief Scientist and Head (VP) of Global Pathogen Safety of Product Supply-Biologics of Bayer Pharmaceuticals, discussed the hopes and challenges in gene therapy. He talked about the history of gene therapy, current ongoing over 3,000 clinical trials, global market, as well as regulation and reimbursement issues. He emphasized the challenges of gene therapy such as purification efficiency, and manufacturing capacity but also reminded the audience of all the opportunities when we entered the modern era of gene therapy.
We would like to express our gratitude to Dr. Shengjiang Liu, Dr. Gary Li, Dr. Haifeng Chen, Dr. Niren Murthy and Dr. Kee-Hong Kim for their excellent presentations.
We are very thankful for CABS volunteers including Dr. Xiang Yi, Dr. Ken Zhang, Dr. Hao Shao, Ms. Xu Chen, Dr. Hesong Han, Dr. Furong Yu, Dr. Xuefeng Wang, Dr. Qiang Gan, Ms. Ruowei Liu, Dr. Xiangle Sun, and Ms. Jing Chen for their great volunteer works including the event organization and logistics.
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