公开(公告)号：WO2020023612A1

公开(公告)日：2020-01-30

申请号：PCT/US2019/043196

申请日：2019-07-24

申请人： 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

IPC分类号： A61P25/14 ,  C12N15/86

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.

公开(公告)号：WO2021154923A2

公开(公告)日：2021-08-05

申请号：PCT/US2021/015393

申请日：2021-01-28

申请人： VOYAGER THERAPEUTICS, INC. ,  MORRISON, Christopher J.

发明人： MATHUR, Krishanu ,  HENDRICKS, Andrade ,  LUTHER, Matthew ,  CARDINAL, Jacob J. ,  HURWIT, Daniel S. ,  KARPES, Lori B.

IPC分类号： C12N15/86 ,  C12N15/864 ,  C12N15/866 ,  C12N7/02 ,  A61K48/00 ,  C12N2710/14143 ,  C12N2710/14144 ,  C12N2750/14143 ,  C12N2750/14151

摘要： The present disclosure describes methods and systems for use in the production of recombinant adeno-associated virus (rAAV) particles comprising a payload (e.g., a polynucleotide encoding aromatic L-amino acid decarboxylase (AADC) or a functional variant thereof). In certain embodiments, the production process uses Sf9 insect cells as viral production cells. In certain embodiments, the production process and system use Baculoviral Expression Vectors (BEVs) and/or Baculoviral Infected Insect Cells (BIICs) in the production of rAAV particles.

公开(公告)号：WO2020150556A1

公开(公告)日：2020-07-23

申请号：PCT/US2020/014000

申请日：2020-01-17

申请人： VOYAGER THERAPEUTICS, INC.

发明人： MARANGA, Luis ,  MORRISON, Christopher J. ,  MATHUR, Krishanu ,  LUTHER, Matthew ,  HURWIT, Daniel S. ,  CARDINAL, Jacob J. ,  KARPES, Lori B. ,  ANSONDARIA, Aditya ,  FORSTER, James ,  DISMUKE, David ,  STEININGER, Robert

IPC分类号： A61P25/14 ,  C12N15/86

摘要： The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles, comprising recombinant adeno-associated virus (rAAV) particles. In certain embodiments, the production process and system use Sf9 insect cells as viral production cells. In certain embodiments, the production process and system use Baculoviral Expression Vectors (BEVs) and Baculoviral Infected Insect Cells (BIICs) in the production of AAV particles.