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2025 White Paper on Recent Issues in Bioanalysis: Redosing Patients with AAV Gene Therapy; CRS Immunogenicity Risk; Shedding Assays; NHP Studies Immunogenicity; CMC vs Bioanalytical Assays; Artificial Intelligence-Powered Genomic Pipelines for NGS (PART 3A-Recommendations on Gene, Cell, and Vaccine Therapies Immunogenicity & Technologies; Biotherapeutics & Biosimilars Immunogenicity Assessment & Clinical Relevance PART 3B-Regulatory Agencies Input on Immunogenicity/Technologies of Biotherapeutics, Gene, Cell & Vaccine Therapies) 2025年生物分析近期问题白皮书：AAV基因治疗患者的重复给药；CRS免疫原性风险；脱落检测；非人灵长类动物研究免疫原性；CMC与生物分析检测；人工智能驱动的基因组NGS分析流程（第3A部分—基因、细胞和疫苗治疗免疫原性及技术的建议；生物药与生物类似药免疫原性评估及临床相关性；第3B部分—监管机构对生物药、基因、细胞及疫苗治疗免疫原性/技术的意见） Tounekti, Omar; Shubow, Sophie; Wassmer, Sarah; Van Tuyl, Andrea; Yang, Lin; Gong, Binsheng; Abhari, Mohsen Rajabi; Kalina, Warren; Myler, Heather; DelCarpini, Jason; Escobar, David; Fiscella, Michele; Gorovits, Boris; Gupta, Swati; Irvin, Susan C.; Irwin, Chad; Jankowski, Wojciech; Jawa, Vibha; Karauzum, Hatice; Kerridge, Claire; Lu, Yanmei; Mayer, Christian; Mcgregor, Jessica; Melton, Andrew; Murphy, Rocio; Nanra, Jasdeep; Nowocin, Anna; Partridge, Michael A.; Prior, Sandra; Sauna, Zuben; Scott, Bradley; Seitzer, Jessica; Stern, Mark; Sugimoto, Hiroshi; Taylor, Melissa; Wong, Adrian; Xiao, Wenming; Xu, Yuanxin; Yang, Tong-Yuan; Yuill, Daisy; Kubiak, Robert J.; Thway, Theingi; Gunn, George; Pelto, Ryan; Sumner, Giane; Baltrukonis, Daniel; Dholakiya, Sanjay L.; Dogmanits, Shannon; Garofolo, Fabio; Jadhav, Maria; Kramer, Daniel; Meyer, Erik; Mohapatra, Susovan; Mozaffari, Reza; Peng, Kun; Pine, Samuel; Poetzl, Johann; Rasamoelisolo, Michele; Shao, Weiping; Staack, Roland F.; Swanson, Steven; Taqui, Atiya; Vauleon, Stephanie; Wang, Yun; Wen, Yi 分享 收藏
CAMPSIITET Phase I/II/III: An interim clinical study update of RGX-121, an investigational gene therapy for the treatment of neuronopathic mucopolysaccharidosis type II (MPS II) Pisani, Laura; Ficicioglu, Can; Harmatz, Paul; Giugliani, Roberto; Rajan, Deepa; Hagood, Joseph; Fiscella, Michele; Yang, Lin; Gilmor, Michelle; Cho, Yoonjin; Mulatya, Caroline; Phillips, Dawn; Boulos, Nidal; Falabella, Paulo 分享 收藏
CAMPSIITETM phase I/II/III: An interim clinical study update of RGX-121, an investigational gene therapy for the treatment of neuronopathic mucopolysaccharidosis type II (MPS II) Harmatz, Paul; Ficicioglu, Can; Giugliani, Roberto; Rajan, Deepa; Hagood, Joseph; Fiscella, Michele; Yang, Lin; Gilmor, Michelle; Cho, Yoonjin; Mulatya, Caroline; Phillips, Dawn; Boulos, Nidal; Falabella, Paulo; Pisani, Laura 分享 收藏
DeePhys: A machine learning-assisted platform for electrophysiological phenotyping of human neuronal networks Hornauer, Philipp; Prack, Gustavo; Anastasi, Nadia; Ronchi, Silvia; Kim, Taehoon; Donner, Christian; Fiscella, Michele; Borgwardt, Karsten; Jagasia, Ravi; Taylor, Verdon; Roqueiro, Damian; Hierlemann, Andreas; Schroter, Manuel 分享 收藏
Evaluation of Cellular Immune Response to Adeno-Associated Virus-Based Gene Therapy 腺相关病毒基因治疗的细胞免疫应答评价 Gorovits, Boris; Azadeh, Mitra; Buchlis, George; Fiscella, Michele; Harrison, Travis; Havert, Mike; Janetzki, Sylvia; Jawa, Vibha; Long, Brian; Mahnke, Yolanda D.; McDermott, Andrew; Milton, Mark; Nelson, Robert; Vettermann, Christian; Wu, Bonnie 分享 收藏
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RGX-121 gene therapy for the treatment of neuronopathic mucopolysaccharidosis type II (MPS II): Interim analysis of data from the first in human study Harmatz, Paul; Ficicioglu, Can H.; Giugliani, Roberto; Rajan, Deepa; Hagood, Joseph; Fiscella, Michele; Yang, Lin; Gilmor, Michelle; Cho, Yoonjin; Phillips, Dawn; Falabella, Paulo 分享 收藏
RGX-111 gene therapy for the treatment of severe mucopolysaccharidosis type I (MPS I): Interim analysis of data from the first in human study Wang, Raymond Y.; Ficicioglu, Can H.; Giugliani, Roberto; Burke, Jenna; Fiscella, Michele; Yang, Lin; Gilmor, Michelle; Cho, Yoonjin; Phillips, Dawn; Falabella, Paulo 分享 收藏
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Identification of a biomarker that differentiates neuronopathic forms of MPS I and MPS II Boulos, Nidal; Wu, Zhuchun; Cho, Yoonjin; Mulatya, Caroline; Fuller, Maria; Ausseil, Jerome; Pavone, Luigi M.; Giugliani, Roberto; Fiscella, Michele; Nevoret, Marie-Laure 分享 收藏
RGX-121 gene therapy for the treatment of severe mucopolysaccharidosis type II (MPS II): Interim analysis of data from the first in-human study Harmatz, Paul; Escolar, Maria; Ficicioglu, Can; Giugliani, Roberto; Nevoret, Marie-Laure; Fiscella, Michele; Yang, Lin; Cho, Yoonjin; Phillipse, Dawn; Falabella, Paulo 分享 收藏
Subretinal injection of RGX-381 to cynomolgus monkeys leads to supraphysiological levels of TPP1 in the eye Buss, Nicholas; Budzynski, Ewa; Yang, Lin; Kim, SunJung; Kim, Kwi Hye; Higgins, Mikayla; Gardner, Louis; Fiscella, Michele; Poole, Timothy A.; Wang, Sophie H.; Nelvagal, Hemanth R.; Cooper, Jonathan D.; Danos, Olivier 分享 收藏
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Versatile live-cell activity analysis platform for characterization of neuronal dynamics at single-cell and network level Yuan, Xinyue; Schroter, Manuel; Obien, Marie Engelene J.; Fiscella, Michele; Gong, Wei; Kikuchi, Tetsuhiro; Odawara, Aoi; Noji, Shuhei; Suzuki, Ikuro; Takahashi, Jun; Hierlemann, Andreas; Frey, Urs 分享 收藏
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AAV8-vectored suprachoroidal gene transfer produces widespread ocular transgene expression Ding, Kun; Shen, Jikui; Hafiz, Zibran; Hackett, Sean F.; Silva, Raquel Lima e; Khan, Mahmood; Lorenc, Valeria E.; Chen, Daiqin; Chadha, Rishi; Zhang, Minie; Van Everen, Sherri; Buss, Nicholas; Fiscella, Michele; Danos, Olivier; Campochiaro, Peter A. 分享 收藏
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