公开(公告)号：WO2020077165A1

公开(公告)日：2020-04-16

申请号：PCT/US2019/055756

申请日：2019-10-11

申请人： VOYAGER THERAPEUTICS, INC.

发明人： MURLIDHARAN, Giridhar ,  SAH, Dinah Wen-Yee ,  PATZKE, Holger ,  CARTER, Todd ,  HOU, Jinzhao ,  ADACHI, Kei ,  REN, Amy

IPC分类号： C07K14/005 ,  C12N15/86

摘要： The disclosure provides compositions and methods for the preparation, manufacture, formulation and therapeutic use of adeno-associated virus (AAV) particles for the prevention and/or treatment of diseases.

公开(公告)号：WO2020033926A9

公开(公告)日：2020-02-13

申请号：PCT/US2019/046061

申请日：2019-08-09

申请人： COMPASS THERAPEUTICS LLC

发明人： BOBROWICZ, Piotr ,  WIDBOOM, Paul ,  SCHMIDT, Michael, March

IPC分类号： A61P35/00 ,  C07K16/28 ,  A61K39/00

摘要： The present disclosure relates to, inter alia, compounds (e.g., antibodies, or antigen-binding fragments thereof) that bind to CD277, for treating, or ameliorating one or more symptoms of cancer.

公开(公告)号：WO2020028751A3

公开(公告)日：2020-02-06

申请号：PCT/US2019/044796

申请日：2019-08-02

申请人： VOYAGER THERAPEUTICS, INC. ,  CALIFORNIA INSTITUTE OF TECHNOLOGY

发明人： SAH, Dinah Wen-Yee ,  HOU, Jinzhao ,  ADACHI, Kei ,  WANG, Wei ,  CHEN, Qingmin ,  CARROLL, Jenna ,  REN, Amy ,  REN, Xiao-Qin ,  DEVERMAN, Benjamin E. ,  GRADINARU, Viviana ,  HUANG, Qin

IPC分类号： C07K14/005 ,  C12N15/86

摘要： The disclosure relates to compositions, methods, and processes for the preparation, use, and/or formulation of adeno-associated virus capsid proteins, wherein the capsid proteins comprise targeting peptide inserts for enhanced tropism to a target tissue.

公开(公告)号：WO2019222328A1

公开(公告)日：2019-11-21

申请号：PCT/US2019/032384

申请日：2019-05-15

申请人： VOYAGER THERAPEUTICS, INC.

发明人： RAVINA, Bernard ,  KELLS, Adrian Philip

IPC分类号： C12N15/62 ,  A61K38/51 ,  A61K48/00 ,  C12N15/86

摘要： The present disclosure relates to methods, formulations and devices for the delivery and therapeutic administration of polynucleotides encoding AADC. The present disclosure relates to methods, formulations and devices for the delivery and therapeutic administration of AAV vectors which include polynucleotides encoding AADC. The present disclosure relates to methods, formulations and devices for the delivery7 and therapeutic administration of polynucleotides encoding AADC in the treatment of neurological diseases, disorders and conditions, including Parkinson's Disease.

公开(公告)号：WO2019195386A1

公开(公告)日：2019-10-10

申请号：PCT/US2019/025526

申请日：2019-04-03

申请人： TARVEDA THERAPEUTICS, INC.

发明人： BILODEAU, Mark T. ,  ZHOROV, Eugene ,  SHINDE, Rajesh R. ,  SEARS, Christopher ,  BRADY, Samantha ,  SWERYDA-KRAWIEC, Beata

IPC分类号： A61K9/00 ,  A61K9/14 ,  A61K9/19 ,  A61K47/00 ,  A61K47/06 ,  A61K47/60

摘要： The present invention provides improved formulation with reduced tert-butanol levels and/or manufacturing process for drug compounds that form a solvate with tertbutanol. For example, the drug compound may be SDC-TRAP-0063.

公开(公告)号：WO2019195384A1

公开(公告)日：2019-10-10

申请号：PCT/US2019/025524

申请日：2019-04-03

申请人： TARVEDA THERAPEUTICS, INC.

发明人： BILODEAU, Mark T. ,  MOREAU, Benoît ,  KADIYALA, Sudhakar ,  WOOSTER, Richard ,  WHITE, Brian H.

IPC分类号： A61K38/16 ,  A61K39/395 ,  A61K45/00 ,  A61P43/00

摘要： Conjugates of an active agent attached to a targeting moiety, such as at least one HSP90 binding moiety, via a linker, have been designed. Such conjugates can provide improved temporospatial delivery of the active agent, improved biodistribution and penetration in tumor, and/or decreased toxicity. Methods of making the conjugates and the formulations thereof are provided. Methods of administering the formulations to a subject in need thereof are provided, for example, to treat or prevent cancer.

公开(公告)号：WO2019173434A1

公开(公告)日：2019-09-12

申请号：PCT/US2019/020892

申请日：2019-03-06

申请人： VOYAGER THERAPEUTICS, INC.

发明人： HOROWITZ, Eric D. ,  CECCHINI, Sylvain ,  STEININGER, Robert ,  DISMUKE, David ,  MORRISON, Christopher J.

IPC分类号： C12N15/864

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：WO2019051436A1

公开(公告)日：2019-03-14

申请号：PCT/US2018/050317

申请日：2018-09-11

申请人： RA PHARMACEUTICALS, INC.

发明人： FAHRNER, Richard Lee ,  PHILLIPS, Christian Winthrop

IPC分类号： C12P7/64

CPC分类号： A61K9/0019 ,  A61K9/08 ,  A61K47/14

摘要： The present disclosure provides formulations for sustained release of therapeutic agents. Included are formulations incorporating a mixture of acylglycerols. Also included are methods of delivering therapeutic agents to subjects using sustained release formulations and methods of treating complement-related indications using formulations disclosed.

公开(公告)号：WO2019036430A1

公开(公告)日：2019-02-21

申请号：PCT/US2018/046634

申请日：2018-08-14

申请人： CAMP4 THERAPEUTICS CORPORATION

发明人： BUMCROT, David A. ,  SEHGAL, Alfica ,  SIGOVA, Alla ,  SCHWARTZ, Brian Elliot ,  WHISSELL, Gavin

IPC分类号： C12Q1/44 ,  C12N9/16 ,  C12N9/20

摘要： The present invention provides methods and compositions for the treating a patient with one or more conditions associated with PNPLA3, such as nonalcoholic fatty liver disease (NAFLD), nonalcoholic steatohepatitis (NASH), and/or alcoholic liver disease (ALD). Methods and compositions are also provided for modulating the expression of the PNPLA3 gene in a cell by altering gene signaling networks.

公开(公告)号：WO2018231759A1

公开(公告)日：2018-12-20

申请号：PCT/US2018/037005

申请日：2018-06-12

申请人： OBSIDIAN THERAPEUTICS, INC.

发明人： SURI, Vipin ,  DELABARRE, Byron ,  GLADSTONE, Michael, N. ,  RICHARDSON, Celeste ,  DOLINSKI, Brian ,  KULKARNI, Abhishek ,  INNISS, Mara, Christine ,  SUN, Dexue ,  LI, Dan, Jun ,  OLINGER, Grace, Y. ,  HELLER, Scott, Francis

IPC分类号： A61K38/17 ,  A61K39/39 ,  C07K16/28 ,  C07K19/00

摘要： The present invention relates to compositions and methods for the regulated and controlled expression of proteins. Methods for inducing anti-cancer immune responses in a subject are also provided.