公开(公告)号：US11406690B2

公开(公告)日：2022-08-09

申请号：US16588130

申请日：2019-09-30

Applicant: BIOMARIN PHARMACEUTICAL INC. ,  UCL BUSINESS PLC ,  ST. JUDE CHILDREN'S RESEARCH HOSPITAL

Inventor： Peter Cameron Colosi ,  Amit Nathwani ,  Jenny McIntosh ,  Edward Tuddenham ,  Andrew Davidoff

IPC: A01N63/00 ,  A61K38/37 ,  A61K48/00 ,  C12N7/00 ,  C07K14/755 ,  C12N15/86

Abstract: The present disclosure provides methods of generating multiplexed genetically modified animals, for example, porcine endogenous retrovirus (PERV)-inactivated pigs. The disclosure also provides methods of improving the birth rate of multiplexed genetically modified animals. In some embodiments, the present closure is concerned with the generation and utilization of porcine cells in which porcine endogenous retroviral (PERV) elements have been inactivated. In sonic embodiments, the PERV-free or PERV-reduced porcine cells are cloned to produce porcine embryos. In some embodiments, the PERV-free or PERV-reduced embryos may be grown into adult swine from which organs and/or tissues may be extracted and used for such purposes as xenotransplantation into non-porcine animals such as humans.

公开(公告)号：US20220136051A1

公开(公告)日：2022-05-05

申请号：US17432721

申请日：2020-02-24

Applicant: St. Jude Children's Research Hospital

Inventor： Yiping Fan ,  Jeremy Crawford ,  Benjamin Youngblood

IPC: C12Q1/6881 ,  G16B40/20

Abstract: Provided herein are methods and compositions for determining T-cell differentiation by comparing the methylation status of T cells relative to a T cell methylation index and using this determination to identify or isolate populations of T cells having desired T cell multipotency. Further, the present methods and compositions can be used to monitor or treat symptoms of chronic infections, autoimmune diseases, and cancer.

公开(公告)号：US20210369869A1

公开(公告)日：2021-12-02

申请号：US17401765

申请日：2021-08-13

Applicant: ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC.

Inventor： John T. GRAY

IPC: A61K48/00 ,  A61K35/76 ,  C12N15/86 ,  C12N7/00 ,  C12N15/861

Abstract: The present invention relates to nucleic acid molecules containing spacers and methods of using the same.

公开(公告)号：US11116757B2

公开(公告)日：2021-09-14

申请号：US16683076

申请日：2019-11-13

Applicant: Memorial Sloan Kettering Cancer Center ,  St. Jude Children's Research Hospital

Inventor： Jaeki Min ,  Daniel C. Scott ,  Deepak Bhasin ,  Brenda A. Schulman ,  Bhuvanesh Singh ,  Jared T. Hammill ,  R. Kiplin Guy

IPC: A61K31/4545 ,  C07D401/12 ,  C07D211/58 ,  C07D401/06 ,  C07D409/14 ,  C07D405/12 ,  C07D417/06 ,  C07D471/04 ,  C07D405/06 ,  C07D401/04 ,  C07D417/04 ,  A61P31/12 ,  A61P15/16 ,  A61P31/04 ,  A61K31/17 ,  A61K31/437 ,  A61K31/451 ,  A61K31/4525 ,  A61K31/454 ,  A61K31/506

Abstract: In one aspect, the invention relates to substituted 1-phenyl-3-(piperidin-4-yl)urea analogs, derivatives thereof, and related compounds, which are useful as inhibitors of the DCN1-UBC12 interaction inhibitors of DCN1-mediated cullin-RING ligase activity, methods of making same, pharmaceutical compositions comprising same, methods of treating disorders using the disclosed compounds and compositions, methods of treating disorders associated with a DCN1-UBC12 interaction dysfunction, methods of treating disorders associated with a DCN1-mediated cullin-RING ligase activity dysfunction, methods of male contraception comprising the disclosed compounds and compositions, and kits comprising the disclosed compounds and compositions. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.

公开(公告)号：US10894061B2

公开(公告)日：2021-01-19

申请号：US15747902

申请日：2016-08-05

Applicant: Regents of the University of Minnesota ,  St. Jude Children's Research Hospital, Inc. ,  Albert-Ludwig Universität Freiburg ,  The University of North Carolina at Chapel Hill

Inventor： Bruce R. Blazar ,  Brent Koehn ,  Peter J. Murray ,  Jenny P. Y. Ting ,  Robert Zeiser ,  Jeff S. Miller

IPC: A01N63/00 ,  A61K48/00 ,  A61K35/15 ,  C12N5/0787 ,  C12N5/078 ,  A61P37/06 ,  A61K39/00 ,  A61K45/06 ,  C12N5/0786 ,  A61K35/12

Abstract: In one aspect, a method of treating a subject having or at risk of having graft-versus-host disease (GvHD) generally includes administering to the subject a plurality of myeloid-derived suppressor cells (MDSCs) effective to ameliorate at least one symptom or clinical sign of graft-versus-host disease compared to a suitable control subject. In another aspect, a method of treating a tumor in a subject generally includes administering to the subject an anti-tumor therapy and co-administering to the subject an inflammasome inciting agent in an amount effective to increase inflammasome activation of MDSCs sufficiently to reduce suppressor function of the MDSCs.

公开(公告)号：US10801012B2

公开(公告)日：2020-10-13

申请号：US15857147

申请日：2017-12-28

Applicant: ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC. ,  NATIONAL UNIVERSITY OF SINGAPORE

Inventor： Dario Campana ,  Yu-Hsiang Chang

IPC: A61K35/17 ,  C12N5/0783 ,  A61K38/00 ,  C07K14/725 ,  C07K14/705 ,  C12N5/00

Abstract: The invention provides a chimeric receptor comprising NKG2D, DAP10 and CD3 zeta. Also disclosed is a composition comprising this chimeric receptor and methods for making and using it to enhance the cytotoxicity and antitumor capacity of NK cells. The invention also encompasses methods for use of NKG2D-DAP10-CD3 zeta polypeptides, vectors and cells in methods for treating cancer and other proliferative disorders, as well as infectious diseases.

公开(公告)号：US20200318197A1

公开(公告)日：2020-10-08

申请号：US16719497

申请日：2019-12-18

Applicant: STC.UNM ,  ST. JUDE CHILDREN'S RESEARCH HOSPITAL ,  THE CHILDREN'S HOSPITAL OF PHILADELPHIA on behalf of CHILDREN'S ONCOLOGY GROUP

Inventor： Cheryl L. Willman ,  Stephen P. Hunger ,  Charles Mullighan ,  I-Ming Chen ,  Kathryn G. Roberts ,  Huining Kang ,  Richard C. Harvey

IPC: C12Q1/6886

Abstract: The invention provides arrays, systems, devices, methods, computer-readable media and kits that enable expression-based classification of B-precursor acute lymphoblastic leukemia (ALL) as being either responsive or non-responsive to tyrosine kinase inhibitor mono or co-therapy.

公开(公告)号：US10676510B2

公开(公告)日：2020-06-09

申请号：US15863673

申请日：2018-01-05

Applicant: ST. JUDE CHILDREN'S RESEARCH HOSPITAL

Inventor： Richard E. Lee ,  Ying Zhao ,  Elizabeth Griffith ,  Zhong Zheng ,  Aman P. Singh

IPC: C07K11/02 ,  A61K38/15 ,  A61K45/06 ,  C12N9/50 ,  C07K7/06 ,  A61K38/00

Abstract: In one aspect, the invention relates to substituted urea depsipeptide analogs, derivatives thereof, and related compounds, which are useful as activators the C1pP endopeptidease; synthesis methods for making the compounds; pharmaceutical compositions comprising the compounds; and methods of treating infectious disease using the compounds and compositions. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.

公开(公告)号：US20200079739A1

公开(公告)日：2020-03-12

申请号：US16468657

申请日：2017-12-12

Applicant: St. Jude Children's Research Hospital

Inventor： R. Kip Guy ,  P. Jake Slavish ,  William Robert Shadrick ,  Brandon M. Young ,  Vincent A. Boyd ,  Nagakumar Bharatham ,  Jeanine E. Price ,  Anang Shelat

IPC: C07D215/42 ,  C07D215/44 ,  C07D405/12 ,  C07D401/12 ,  C07D413/12 ,  C07D401/10 ,  C07D405/10 ,  C07D409/10 ,  C07D215/46

Abstract: In one aspect, compounds and compositions that modulate a bromodomain and methods of making and using same are disclosed. The disclosed compounds and compositions can be useful for disorders associated with inhibition of a bromodomain such as, for example, cancer. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.

公开(公告)号：US20200054660A1

公开(公告)日：2020-02-20

申请号：US16467358

申请日：2017-12-07

Applicant: St. Jude Children's Research Hospital

Inventor： Benjamin Youngblood ,  Hossam Abdelsamed ,  Hazem Ghoneim

IPC: A61K31/7068 ,  C12N5/0783 ,  C12Q1/6827 ,  A61K35/17 ,  A61K45/06 ,  A61P31/14

Abstract: Provided herein are methods and compositions for modulating T-cell activity by altering DNA methylation status. Altering the methylation status of CD8+ T cells can prevent T-cell exhaustion and maintain effector functions during sustained antigen exposure. The methods and compositions can be used to treat symptoms of chronic infections and cancer. Further, the methods and compositions relate to predicting T-cell activity by measuring the methylation status of specific memory cell methylation markers and using the markers to identify and separate populations of CD8 T cell having desired T cell activity. The memory cell methylation markers can further be used to identify subjects with chronic infections or cancer that would benefit from personalized therapy, including immune checkpoint blockade therapy.