Casey A. Maguire

Casey A. Maguire, Ph.D., is Associate Professor of Neurology at Harvard Medical School and Principal Investigator in the Department of Neurology at Massachusetts General Hospital, where he leads a laboratory focused on developing more potent and safer gene delivery systems. He is Co-Founder and Advisor of Chameleon Biosciences and Co-Founder and Consultant of Skylark Bio. Dr. Maguire is internationally recognized as the principal inventor of exosome-associated AAV (exo-AAV) technology, a breakthrough platform that addresses critical challenges in AAV gene therapy: pre-existing neutralizing antibodies and biological barrier penetration. Exo-AAV vectors utilize lipid-based nanoparticles (exosomes) secreted by cells to envelope AAV particles, enabling immune evasion, enhanced blood-brain barrier crossing, targeted delivery, and improved safety profiles. His 2017 Molecular Therapy publication demonstrated that exo-AAV successfully delivered genes to inner ear hair cells, restoring hearing in a mouse model of hereditary deafness. This work established the scientific foundation for Skylark Bio's hearing disorder gene therapy pipeline. Maguire has made significant contributions to AAV gene therapy for glioblastoma (GBM). His 2010 Journal of Neuro-Oncology paper described directed evolution of chimeric AAV capsids achieving 97% transduction efficiency in glioma cells through DNA shuffling of six AAV serotypes. His 2016 Molecular Therapy: Oncolytics publication reported the first systemic AAV9-sTRAIL gene therapy for brain tumors, demonstrating tumor growth inhibition and significantly extended survival in patient-derived GBM xenograft models. The Maguire laboratory has published over 50 peer-reviewed papers with more than 6,744 citations. He holds five issued U.S. patents on novel AAV-based gene delivery systems, with core exo-AAV intellectual property licensed to Chameleon Biosciences for their EVADER™ technology platform. His recent work includes developing CRISPR-based therapeutic approaches targeting vascular smooth muscle for multisystemic smooth muscle dysfunction syndrome (MSMDS)—representing the first gene editing therapy specifically designed for the vasculature. He is a funded investigator of Cure Alzheimer's Fund, developing gene delivery vehicles for in vivo Alzheimer's disease therapy.

Casey A. Maguire哲学博士，是哈佛医学院神经学副教授和麻省总医院神经学系首席研究员，领导一个专注于开发更有效和更安全基因递送系统的实验室。他是Chameleon Biosciences的联合创始人兼顾问，以及Skylark Bio的联合创始人兼顾问。 Maguire博士国际公认为外泌体相关AAV (exo-AAV)技术的主要发明者，这是一个突破性平台，解决了AAV基因治疗的关键挑战：预存中和抗体和生物屏障穿透。Exo-AAV载体利用细胞分泌的脂基纳米颗粒（外泌体）包裹AAV颗粒，实现免疫逃避、增强血脑屏障穿越、靶向递送和改善的安全性特征。他2017年在Molecular Therapy上的出版物证明，exo-AAV成功地将基因递送到内耳毛细胞，恢复了遗传性耳聋小鼠模型的听力。这项工作为Skylark Bio的听力障碍基因治疗管线奠定了科学基础。 Maguire在胶质母细胞瘤(GBM) AAV基因治疗方面做出了重要贡献。他2010年在Journal of Neuro-Oncology上的论文描述了通过6个AAV血清型的DNA改组定向进化嵌合AAV衣壳，在胶质瘤细胞中实现97%转导效率。他2016年在Molecular Therapy: Oncolytics上的出版物报告了首个系统性AAV9-sTRAIL脑肿瘤基因治疗，在患者来源的GBM异种移植模型中证明肿瘤生长抑制和显著延长的生存期。 Maguire实验室已发表50多篇同行评审论文，引用超过6,744次。他拥有5项关于新型AAV基因递送系统的已颁发美国专利，核心exo-AAV知识产权授权给Chameleon Biosciences用于其EVADER™技术平台。他最近的工作包括开发针对血管平滑肌的基于CRISPR的治疗方法，用于多系统平滑肌功能障碍综合征(MSMDS)——代表首个专门针对血管系统设计的基因编辑治疗。他是Cure Alzheimer's Fund资助的研究员，为阿尔茨海默病体内治疗开发基因递送载体。