公开(公告)号：US20210355454A1

公开(公告)日：2021-11-18

申请号：US17262271

申请日：2019-07-24

申请人： VOYAGER THERAPEUTICS, INC.

发明人： Jacob J. Cardinal ,  Robert Steininger ,  Lori B. Karpes ,  Christopher J. Morrison ,  Daniel S. Hurwit ,  Matthew Luther ,  Andrew M. Wood ,  Dinah Wen-Yee Sah ,  Pengcheng Zhou ,  Jeffrey S. Thompson ,  Christina Gamba-Vitalo ,  Jenna Carroll Soper ,  Steven M. Hersch ,  Todd Carter

IPC分类号： C12N7/02 ,  C12N15/86 ,  A61K35/76 ,  A61P25/14

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 neurodegenerative disease (e.g., Huntington's Disease (HD)) are also disclosed.

公开(公告)号：US20210010028A1

公开(公告)日：2021-01-14

申请号：US16978288

申请日：2019-03-06

申请人： VOYAGER THERAPEUTICS, INC.

发明人： Eric D. Horowitz ,  Sylvain Cecchini ,  Robert Steininger ,  David Dismuke ,  Christopher J. Morrison

IPC分类号： C12N15/86 ,  C12N9/88

摘要： The present disclosure is directed to parvovirus genomes; plasmid vectors encoding parvovirus genomes, and particles and populations thereof; as well as methods of their production and use.

公开(公告)号：US20220064671A1

公开(公告)日：2022-03-03

申请号：US17423737

申请日：2020-01-17

申请人： VOYAGER THERAPEUTICS, INC.

发明人： Luis Maranga ,  Christopher J. Morrison ,  Krishanu Mathur ,  Matthew Luther ,  Daniel S. Hurwit ,  Jacob J. Cardinal ,  Lori B. Karpes ,  Aditya Ansondaria ,  James Forster ,  David Dismuke ,  Robert Steininger

IPC分类号： C12N15/86 ,  A61K48/00

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.