专利名称 | SYSTEMS AND METHODS FOR PRODUCING GENE THERAPY FORMULATIONS | ||
申请号 | WOUS19043196 | 申请日 | |
公开(公告)号 | WO2020023612A1 | 公开(公告)日 | |
申请(专利权)人 | VOYAGER THERAPEUTICS INC | 发明人 | CARDINAL Jacob J; STEININGER Robert; KARPES Lori B; MORRISON Christopher J; HURWIT Daniel S; LUTHER Matthew; WOOD Andrew M; SAH Dinah Wen Yee; ZHOU Pengcheng; THOMPSON Jeffrey S; GAMBA VITALO Christina; CARROLL Jenna; HERSCH Steven M; CARTER Todd |
专利来源 | 国家知识产权局 | 转化方式 | |
摘要 |
The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles and AAV formulations, including recombinant adeno-associated virus (rAAV) particles and formulations. In certain embodiments, the present disclosure presents methods and systems for clarifying, purifying, formulating, filtering and processing AAV particles and AAV formulations. The present disclosure also describes compositions, methods and processes for the design, preparation, manufacture, use and/or formulation of AAV particles comprising modulatory polynucleotides, e.g., polynucleotides encoding small interfering RNA (siRNA) molecules which target the Huntingtin (HTT) gene (e.g., the wild-type or the mutated CAG-expanded HTT gene). Methods for using formulated AAV particles comprising modulatory polynucleotides to inhibit the HTT gene expression in a subject with a eurodegenerative disease (e.g., Huntington' s Disease (HD)) are also disclosed. |
主管部门:海南中小企业服务 | 建设单位:海南商业联合会
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