公开(公告)号：US20210361318A1

公开(公告)日：2021-11-25

申请号：US17257418

申请日：2019-07-02

申请人： VOYAGER THERAPEUTICS, INC.

发明人： Holger Patzke ,  Steven M. Hersch ,  Jenna Carroll Soper ,  Adrian Philip Kells ,  Nicholas Boulis ,  Donna T. Ward

IPC分类号： A61B17/34 ,  A61B90/30 ,  C12N15/113

摘要： The present disclosure relates to cannula systems for delivery of a therapeutic agent to parenchymal tissue of a target subject. In certain embodiments, the cannula system is for delivery of a therapeutic agent to parenchymal tissue in the spine of a target subject. In certain embodiments, the cannula system includes a cannula positioning guide (CPG) useful in the delivery of a therapeutic agent to a localized target tissue of a subject. The guide may be used in a surgical suite or during a surgical procedure.

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：US20220275367A1

公开(公告)日：2022-09-01

申请号：US17629552

申请日：2020-07-24

申请人： VOYAGER THERAPEUTICS, INC.

发明人： Dinah Wen-Yee Sah ,  Pengcheng Zhou ,  Jeffrey S. Thompson ,  Christina Gamba-Vitalo ,  Jenna Carroll Soper ,  Steven M. Hersch ,  Todd Carter ,  Jacob J. Cardinal ,  Lori B. Karpes ,  Bin Liu

IPC分类号： C12N15/113 ,  A61K35/76 ,  A61K47/02 ,  A61K47/26 ,  A61K47/10 ,  A61P25/28

摘要： The present disclosure relates to pharmaceutical compositions of adeno-associated viral (AAV) particles encoding siRNA molecules and methods for treating Huntington's Disease (HD).

公开(公告)号：US20210254103A1

公开(公告)日：2021-08-19

申请号：US17252584

申请日：2019-07-02

申请人： VOYAGER THERAPEUTICS, INC.

发明人： Dinah Wen-Yee Sah ,  Qingmin Chen ,  Jenna Carroll Soper ,  Holger Patzke ,  Jinzhao Hou ,  Steven M. Hersch

IPC分类号： C12N15/86 ,  A61K9/00 ,  C12N7/00 ,  C12N15/113 ,  A61P25/28

摘要： The present disclosure relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and delivery methods for the treatment of spinal cord related disorders including ALS.