James M. Wilson

James M. Wilson, M.D., Ph.D., is CEO of Gemma Biotherapeutics and Franklin Biolabs, companies focused on advancing AAV gene therapies for rare diseases. Until August 2024, Dr. Wilson spent over three decades at the University of Pennsylvania's Perelman School of Medicine, where he served as Director of the Gene Therapy Program, Rose H. Weiss Professor, and Director of the Orphan Disease Center. Dr. Wilson is a pioneering figure in AAV-based gene therapy whose laboratory discovered an entire family of endogenous adeno-associated viruses, including AAV7, AAV8, and AAV9. Most notably, AAV9—discovered around 2002-2004 by Guangping Gao in Wilson's lab—demonstrated the remarkable ability to cross the blood-brain barrier after systemic administration, revolutionizing CNS gene therapy. AAV9 became one of the most widely used vectors in medicine and forms the basis of onasemnogene abeparvovec (Zolgensma), the FDA-approved therapy for spinal muscular atrophy. Beyond vector discovery, Wilson's team has been instrumental in understanding AAV safety and dose-limiting toxicities. His 2018 Human Gene Therapy publication on severe toxicity in nonhuman primates following high-dose AAV administration provided critical insights into innate immune responses, informing safer clinical development across the field. Wilson has founded multiple gene therapy companies including Genovo, Regenxbio, Passage Bio, and Scout Bio. In October 2025, he launched RareTx, focusing on AAV therapies for lysosomal storage disorders. Wilson advocates a "platform" approach: treating groups of similar diseases using the same capsid, route, dose, and manufacturing, varying only the therapeutic gene—a strategy enabling scalable rare disease treatment.

James M. Wilson医学博士、哲学博士，现任Gemma生物治疗公司和Franklin生物实验室首席执行官，专注于推进罕见病AAV基因治疗。2024年8月前，Wilson博士在宾夕法尼亚大学佩雷尔曼医学院工作超过三十年，担任基因治疗项目主任、Rose H. Weiss教授和孤儿病中心主任。 Wilson博士是AAV基因治疗的先驱人物，他的实验室发现了整个内源性腺相关病毒家族，包括AAV7、AAV8和AAV9。最值得注意的是AAV9（约2002-2004年由Wilson实验室的Guangping Gao发现），它具有系统给药后穿越血脑屏障的非凡能力，彻底革新了中枢神经系统基因治疗。AAV9成为医学中使用最广泛的载体之一，是FDA批准的脊髓性肌萎缩症治疗药物onasemnogene abeparvovec (Zolgensma)的基础。 除了载体发现，Wilson团队在理解AAV安全性和剂量限制性毒性方面发挥了重要作用。他2018年发表在Human Gene Therapy上的关于非人灵长类动物高剂量AAV给药严重毒性的论文，为先天免疫反应提供了关键见解，指导了整个领域更安全的临床开发。 Wilson创建了多家基因治疗公司，包括Genovo、Regenxbio、Passage Bio和Scout Bio。2025年10月，他推出RareTx，专注于溶酶体储存疾病的AAV治疗。Wilson倡导"平台"方法：使用相同的衣壳、途径、剂量和制造工艺治疗一组相似疾病，仅改变治疗基因——这是实现罕见病规模化治疗的策略。